Cell Therapy Blog

Cell and Gene Therapy Industry - Source Material Survey

noreply@blogger.com (Lee Buckler) — Thu, 03 Feb 2022 04:59:00 +0000

In a survey of >25 US blood centers' annual donor data 2010-2020 inclusive, donations were down in all age groups <55 (even after being normalized for COVID impacts) and donations in most age group were down 21-64%.

Access to large numbers of geographically dispersed and ethnically diverse donors of human blood-derived cells and cellular material of all types (healthy, sick, rare, etc) for use in R&D, testing, manufacturing reagents, and human therapeutics has been described, by some, as critical for the continued evolution of the cell and gene therapy industry.

We aim to gather and share intel on this potential emerging issue. Please participate in this survey to power sector intelligence.

Cell and Gene Therapy Industry Survey

Your participation is much appreciated!

Lesson 2 for Mentees from a Seasoned Career-Changer (a series)

noreply@blogger.com (Lee Buckler) — Sun, 05 Dec 2021 23:58:00 +0000

In this second lesson in my series of 'career-advice-I-wish-I-could-give-myself-30-years-ago', I treat two more popular career platitudes to my own irreverent perspective and take a shot at some of the most common excuses people use for not pursuing the career they wish they had.

Lesson 2 - more platitudes and a kick in the pants

I'm a big fan of having goals in mind. Having said that, while you can plan your next move, please don't even bother to pretend you can 'plan your career'. Okay some people make it look like you can (see my friend Larry from Lesson 1) but really all he did was set himself a goal, plan his 'next move' coming out of graduate school and - voila - that resulted in him having met his goal and determined his entire career (but that's also only true because 30 years later he wakes up every day and still wants it more than any other career). You don't have a magic periscope which allows you to see around the next corner let alone into the next decade so don't pretend you can plan for it or that your future self would even be happy with you if you could and did. What you should have a good sense of, however, is what you want now, what you want next, and what kinds of skills and experience would help get you there.

All good things come to those who wait (but not for those who wait around)

Please don't wish for your dream job to be handed to you out of school. You don't deserve it, you haven't earned it and 99 times out of 100 you'll suck at it. If you're still in school, the first thing you'll learn when you get into your first job is that you learned almost nothing worth knowing in school. Don't get me wrong. I'm a huge proponent of school. It's one of the most critical things you'll do but it certainly isn't the most important and definitely not the most educational. What it does give you, hopefully - aside from that door-opener degree you need - is a way to process information, a way of looking at things, a lens, and some basic tools. You'll walk into that first job and realize you have to learn almost everything immediately critical to surviving the job - none of which you were given in school. So... patience my young Obi-Wan Kenobi. You too shall learn from the masters and be handed the keys when you've earned them. Work hard. Listen harder. Plan a little. Execute a lot. And bide your time.

Patience is a funny thing. On the one hand, it is a virtue. Pushing things to happen your way on your timing almost never works out as well letting things evolve more organically. In part this is because people are way more accepting and supportive of things which they consider to be - at least in part - their idea rather than things foisted on them. On the other hand, sitting around waiting for the world to hand you what you want, when you want it, how you want it will NEVER work out.

So somewhere in between - as usual - is the right balance (note: the world almost always favours balance). Patient but planned persistence. In other words, know what you want, plan for it, work toward it, put it out there and trust that it'll happen when it is right. I won't say "when its meant to be" as if the universe has destined your fate but I do mean that things tend to "just fall in place" when they are a natural fit for all involved rather than when you want them to happen.

For the most part, jobs are given to those who earn them but not necessarily those that deserve them. You may deserve a job - you may have all the qualifications and more - but not be offered it because you didn't earn it. You may earn a job you don't really deserve. To earn a job involves people skills - its about more than you can put on paper, more than your credentials or accomplishments. It's the soft skills that can leapfrog you into places you don't belong. You've got to check some boxes but box-checking alone may leave you stranded in a job well below what you deserve but for your inability to 'earn' a better one.

Almost every job I've been offered feels like it fell into my lap. It didn't. I positioned myself to be offered it, made it clear I wanted it - maybe even applied for it - but is was suddenly just 'there' either because I was actively recruited or because it just seemed like a perfectly natural fit to all around. Not because I'm a rock star or "all that" or was even particularly qualified (on paper) for the job but because by the time it came open, I was considered at least a good - if not an obvious - candidate for the position in the eyes of the decision-makers. Many times that's because I let myself in the side door and bided my time positioning myself for and earning my way into the job I really wanted -- but let me come back to that.

I remember the day in 2000 when I thought to myself for the first time that it would be cool one day to the CEO of a biotech company. At the time it was a ridiculously audacious thought. I had no business background, no science education and I literally couldn't even pronounce half the terminology on my screen on a daily basis.

I didn't myopically focus on that goal. I didn't obsess about it. What I did do was two things. Firstly I tried to take jobs that seemed like they might be on the right path toward that goal and get me one step closer. Secondly, I tried to be painfully honest with myself about what I lacked to one day be offered that job and either fill that gap or compensate for my deficiencies in ways that were accessible to me. Let me give some examples.

Long before my vision for joining the biotech C-suite, I realized I was embarrassingly deficient in most things related to basic business and capital markets. I decided if I read anything every day it would be the business section of the daily newspaper. At first it was like trying to read hieroglyphics but eventually through stubborn osmosis I gradually started to absorb some basic concepts and also define questions I could ask friends, colleagues and mentors who I knew had the answers.

Later, when I found myself surrounded by brilliant pioneers of stem cell research and cell therapy, I found myself ignorant of all things biotech so I began to (a) find ways to immerse myself in meetings and interactions where I could learn and occasionally ask a dumb question and (b) consume every piece of accessible literature on stem cell/cell therapy basics I could and particularly every corporate news release from any company in the sector. I didn't know how that was going to translate into a job opportunity but I knew I wasn't going to get any higher job in biotech without that basic knowledge. I also made it my job to meet and build relationships with people in cell therapy companies in a way that was well beyond my job expectations, in part because I believed doing so would pay off in some way yet to be determined.

That passion for learning and consuming content resulted in the identification of an information gap in the sector which led to the launch of a publication, the information turned into an unparalleled dataset no one else had, and the network turned into a contacts list (rolodex my Dad used to call it). All that resulted in a job offer that relied on a broad (but needn't be deep) understanding of what all the companies were doing in the sector and knowing someone at each of those companies (or someone who knew that someone). That job was business development. It was the perfect job I didn't know I wanted until I had it and it just so happened to be the only job I would be offered with my limited education in science or business. Nonetheless I was actively recruited for the gig because while I didn't deserve it, I had earned it. Interestingly I had been offered a similar position two years earlier but it wasn't the right time and it felt 'forced' by both the potential employer and myself. Two years later it was a perfect fit. Patience rewarded both sides.

Even later in my career, I concluded I was going to hit the glass ceiling of education. Without an advanced science or business degree I determined (rightly or wrongly) that was I unlikely to be able to work my way up to the C-level suite without making my own way there. That was the genesis of my leaving a comfortable mid-level management position to become an entrepreneur and consultant. I would make it or break it on my own and return to the 'work force' when I was offered something closer to the C-suite than I was likely to earn trying to work my up to it as an employee. In retrospect I could likely have earned my way there if I'd been willing to relocate and worked in a big company. I was not. My other priorities dictated - at least heavily influenced - the path I chose.

Back to the side door. The other example of patience is knowing where you want to be even though it involves not being what you want to be for now. For example, when I was looking to jump out of practicing law, I chanced my way - through a friend-of-a-friend - into job interview at a tech company with one of the most interesting men I had ever met in the coolest office I'd ever walked into with one of the greatest views of the city I'd seen. I left thinking if he's crazy enough to give me a job, I'm crazy enough to take it even though I didn't have the faintest idea what they did or what I'd do there. He was crazy, I was crazy and the job I ended up in involved me doing a lot of things no one in their right mind should be doing with a law degree but I was in a milieu I loved and I would have paid to be there. It was the side door to the job I wanted but didn't know existed. I worked my way through, up and around the group of companies - some job offers I deserved, others I earned - until I earned my way out of there six years later into the kind of job many people would have expected to jump into immediately.

That's the 'earn-my-way-up' side door. Another way is the consultant/contactor, part-time or internship side door. If you're trying to punch above your weight class and leapfrog your way into a job for which you barely have the required credentials or experience, you may need to present a potential employer with a low-risk way to determine if you deserve it - just earn your way in. The alternative to the low-level job side door is the 'try-before-you-buy' side door to career leapfrogging. Many a consultant, contractor, part-timer and intern has earned a full-time dream job (or least entrée position to the dream job) by earning their stripes on a test-drive basis.

Be careful what you wish for you just might get it (so be prepared - go in eyes wide open)

I remember the day I was sitting in the high school honors algebra class I had no business trying to pass when I first thought being a teacher might be a good gig because my teacher was pretty cool. Six years later I was standing in front of my own high school class pretending to be infinitely more wise and experienced than kids just a few years younger. They challenged me, tested me and dared me to rise to the occasion or whither in defeat. At one point two of my students were rumoured to have been the subjects of sexually inappropriate behaviour by a teaching colleague and friend. As a young 25-year old teacher I was made aware of the rumour by other students who entrusted me with taking appropriate action - to treat the situation delicately but decisively. Handle this one wrong and it turns out to be false, it ruins the career (and life) of a wonderful teacher; handle it wrong and it turns out to be true, and I only aggravate the potentially devastating travesty done to these children. These are the moments you don't think about when you think to yourself... teaching might be cool.

I've already told you about the day I first thought... maybe I'd like to be a biotech CEO one day. Four months after having been invited to become CEO of a biotech company for the first time, I had to lay off all my staff, sublet the entire office, sell all our furniture, and prepare the company for a complete restructuring. This was not stuff I imagined when I first thought to myself.... that looks like a cool job I would enjoy.

Both of those are a couple of my favourite jobs, hands down, but both brought elements I wouldn't have dared to imagine and certainly never dreamed about. In part, that's why you need hard-earned stripes before you get the job you wish for. You'll need them and they are never the things you learned about in school.

I can't. I don't have time. I'm not qualified. I'm over-qualified. I don't know how. And other lame excuses.

In case you can't tell, I'm not a fan of excuses. They are, after all, just that. Excuses. Most of the time they are not even good reasons. They are fictions we tell ourselves and then say out loud in the belief that they are real based on our experience that they very often are believed and work to relieve us of whatever obligation or opportunity we're trying to escape.

"I can't". Really? Have you seen the videos of the armless, legless motivational speaker or a million other people doing unthinkable things well beyond what is expected to be their capabilities. Rethink what you "can't" do. It's a thinly veiled excuse for I don't want to bad enough and/or I'm too scared to try.

"I don't/didn't have time" is hands down the worst excuse ever invented. You didn't make time. You didn't make time because you had other priorities that you chose to dedicate your time to. If it was so important to you, you would find/make the time. I don't/didn't have time means it wasn't important enough to me to prioritize over the other things taking up my time. I'm not saying you should give up those other things, just be honest about them. If it turns out spending time with your children, or watching football, or gardening, or hanging with your friends is more important than burning the midnight oil getting your executive MBA, that's perfectly okay. Just know it. Say it. But to be perfectly clear, there are single Moms working full time jobs and earning their degree so if you decide not to then it's not because you don't have time it's because you chose to allocate your time differently.

Now to be fair, sometimes there just isn't enough time to get done all that is on your plate. Many times deciding what can be done in the time allotted is not entirely your choice. The point is, never use the "I didn't have time" excuse. Invite those assigning your work to help you prioritize. You are not expected to create time but if it is not your job to assign your own priorities then you are expected to help your superiors or clients determine what you should prioritize in the time allotted. You are expected to not use lack of time as an excuse .. ever. The message here is that effective time management is about managing priorities and the honest excuse is "I didn't manage my priorities properly" rather than "I didn't have time".

"I'm not qualified" ranks right up there with "I can't". Again, literally millions of people are doing things they are not qualified for. I'm one of them.

"I'm over-qualified" simply means your ego won't let you do what you pretend you want to do but clearly don't want to do what it takes to get there.

"I don't know how" is even worse. In this age of content at your fingertips and people accessible with the click of a mouse, there is nothing you can't learn if it is important enough to you to find a way. Wealth isn't even a limiting factor here. Sure you may not be able to get a Harvard education but you can learn how to do almost anything on a shoestring budget if you want it bad enough.

I'm stuck in my job, my mortgage, my marriage, my gender, my life. Not true. To the extent it is true, please just be honest about your role in keeping yourself 'stuck' there. Everything can be changed if you want it bad enough. The only thing you can't change in life is that you have kids. You can pretend you don't have kids but whatever you do - or don't do - doesn't change the fact you have kids. Everything else can be changed. So if it is something other than the fact you have children, if you're unprepared to change it, chances are it's because you don't want it bad enough or at least you don't want it more than the other things preventing you from making that change. I'm not saying you should throw away your comfortable life to make radical change - not at all. I'm just saying you owe it to yourself to be honest about it. If you love the things standing in the way of making a change then admitting that to yourself is freeing. But if you really do want to make a change, you can make the change but it will inevitably involve a shift in priorities. It also involves admitting that the only thing preventing you from making that change you are pretending to want is yourself, your fear and your competing priorities.

Occasionally I'm disappointed because I don't have more money in a #firstworldproblem sort of way but when I'm perfectly honest with myself its because I've never made it a priority. I've chased my interests, my passions, my desire to learn, my hankering for change over money and financial security. I have to acknowledge that those were my choices /my priorities and be comfortable with the results.

Any excuse you can come up with just means that other things are/were more important to you than the thing you're trying to excuse yourself from doing or not doing and that's perfectly okay but please - for the love of humankind - just be honest first with yourself and then the other people around you. Just say you didn't make the time, you are afraid, you're unwilling to do what it takes or to start over, you love the things standing in your way even more than the thing you're pretending to need an excuse for not doing, etc.

So next time you hear yourself making an excuse for not fulfilling an obligation or chasing a dream, challenge yourself to just be honest and face what's really behind that lame excuse.

Lesson 1 for Mentees from a Seasoned Career-Changer (a series)

noreply@blogger.com (Lee Buckler) — Fri, 19 Nov 2021 17:08:00 +0000

I've changed careers too many times and I've left a lot of money on the table but it has been a scintillating, inspiring, and educational adventure.

In this first lesson I've taken three popular platitudes tossed around when giving career advice and given them my own treatment based on my experience and what I wish I could tell myself 30 years ago.

Lesson 1 - the platitudes

Platitudes, like stereotypes, biases, and gut reactions, exist for a reason - because experience and evolution has taught us there is some truth in them but they are not to be relied upon without rational input. Here are my thoughts on three of most common career-focused platitudes.

Follow your passions

I'm convinced almost everyone attempting to give career advice starts with "follow your passions". I won't break the pattern but what I will say is that I think most of the time the underlying assumption is that your career/job should (or would ideally) be aligned with your passion. That's great for those people for whom that is true but I don't think that is necessarily the ideal. You may be one of those people who enjoy their passion being different from what they get paid to do or one those for whom their passion is truly agnostic of a particular career, job, profession or discipline.

Some are blessed knowing their passions very early. A college friend, Larry, knew he wanted to be a professional trumpet player when he was 14 years old. He pursued that single-mindedly and successfully. I was never blessed with that kind of clarity early on. I only put my finger on it in my 40s. Sometimes your passion is conveniently packaged in a career like Larry's was. Sometimes its something quite outside your specific career but very much helps you define it. Sometimes your passion - like mine - is more amorphous and drives you to change careers to fully satisfy it.

My friend Brent's passion is about building financial independence. When he stumbled into a low-level job in a fast-growing company with almost endless upside career opportunity and stock options, he committed his entire career to the company that would be the vehicle to fulling his passion. His particular job - even the company - is simply a means to a very strategic and planned end.

My friends Ron and Mike and Scott share a passion for saving money which drives them to avoid any behavior which risks their savings or ability to add to it. Consequently they stayed in the first secure professional job they got because it gave them the comfort of security with minimal risk and a secure vision for what their future looked like through to and into retirement.

My friend Wayne is passionate about inventing which drove him to engineering school. When he was paid to invent stuff for a big multinational company he loved his job. When they eventually started to stifled his creativity, he did what most inventors do - started inventing his own stuff for his own company. He's not very good at the company stuff and is learning how to surround himself with people who let him invent and they manage the corporate side.

My friend John is passionate about music but refused to ever let himself defined professionally as a musician because he never thought it (or he) was legitimate enough or good enough or whatever his deal was. So he tried his hand at all kinds of things trying to make himself passionate about a career or job until he realized it was outside the workplace in the off-hours when his guitar was in his hands and he was surrounded by music, musicians, and creativity. He also realized he didn't want that to be his job or career - he loved it too much to want to depend on it financially.

My passion is learning. I just love learning and I love the feeling of being a little bit of a 'fish-out-of-water' - being thrown into the deep end of some pool of knowledge and paddling around finding my way to the edges and figuring out how to maneuver in this unknown discipline. I imagined at one point my perfect career path would be as a university professor - surrounded by halls of learning - but I've now come to realize I don't enjoy the narrowing of focus that comes with deep specialization. I want broad, surface-level exposure to a myriad of topics, disciplines, and industries.

Sometimes your passion is aligned with your work; sometimes your work is a means to affording your passion. Neither one is better or lucky or even necessarily more lucrative. What is important is the sooner you can identify your passions and decide how they fit (or not) with your career/job aspirations the sooner you can just get on with it - with being you, pursuing your passions, and living authentically.

Of course there are limit to every such platitude. For example, don't 'follow your passion' if you've decided your passion is a vice or one of the seven deadly sins or illegal. Also, for every 'follow your passion' there are a million examples of where that can lead you astray. Sometimes it can make you sacrifice other things also important to you - like relationships. The insatiable need for balance pervades human existence. Sometimes - left unrestrained or properly channeled - it can lead to more chaos than you want. For example, my passion for learning new things can lead to me just - SQUIRREL (see what I did there) - lacking focus and seeing things through. Decide where your limits are and what other things are important to you that compete with your passion if imbalanced.

Do what you love and you'll be good at what you do

Firstly, this doesn't mean you'll be good at everything you love (see Florence Foster Jenkins). My wife loves music more than I do but she can't sing and never wanted to learn an instrument. She would have made a great radio host with her love of music and great speaking voice. Turns out she loved words even more than music and turned that passion into a career.

Conversely, you won't love everything you're good at. I was very good - at one time - at classical guitar but it never filled my soul. I love singing but I had no desire to do it professionally or to define myself as 'an artist'.

Keep in mind that 'being good at it' does not - at all - mean you'll be financially successful at it. These two things are quite disconnected. The world of commercial success is filled with people who are quite mediocre and, even more so, wonderfully talented people are often impecunious.

For those who are pragmatists, less romantic than I, they might say "love what you do, and you'll be good at it" which to me sounds way to much like resigning to one's fate and having to decide you just love what you do. However, there is some truth to that because even those people dedicate 12 hours per day to their 'love' will sometimes hate it.

Finally, sometime your career is hidden in places your never imagined your passions - or talents - would lead you. Take me, for example. I didn't really like science in grade school. I was fascinated by it much like one is fascinated by something completely foreign to them but I showed no propensity for it and my grades reflected that. Consequently I avoided all things science throughout the rest of my schooling. Now I wake up every day with PHD-envy and find myself happily surrounded by scientists talking science to them. What I discovered is a career in science that didn't require me to be a scientist but allowed be to be constantly fascinated and educated by everything they do. My passion to learn is well fed by immersing myself in a discipline in which I have no business being in based on my education. My passion didn't lead me to be a scientist but my passion led me science.

Okay so maybe I've just ripped this platitude to shreds. If you're not always good at what you love and you won't always love what you're good at, is there any truth to this one? There is.

In every career, in every job, in every company - whether you think so or not - you have options to tweak what you do to match your passions (interests). You may not think so. You do. It may require you moonlighting to prove it but if you're passionate about it, that's no problem. I'll give you an example. I had a job as an association management executive. Many people told me that was my specialty and my job was to focus on the business of association management agnostic of the association's specialty (e.g., trucking, pharmacy, nursing, banking, etc). I disagreed. I found myself passionate about cell therapy and believed that to do the best job possible for the association I was managing I needed to learn as much as a non-scientist could about cell therapy and the challenges the members and industry were facing. That lead me to consuming massive amounts of information which I then decided since I was consuming anyway I might as well share, when lead to the creation of a newsletter, Cell Therapy News, which then lead to hiring a young graduate out of a science journalism program, which lead to the creation of a company that spun out more publications and lead me to blogging and Twitter and LinkedIn Groups, and eventually starting my own cell therapy consulting company and the rest of my career and that young woman I hired is now Vice President in a biotech company. No one asked me to learn as much as I could about cell therapy. No one asked me to launch Cell Therapy News - in fact I had to stay after-hours to build the content and add to the database. But eventually my newsletter caught the attention of the industry and advertisers and my boss and became part of my job and created new jobs.

So "Do what you love" doesn't always come up nicely packaged up for you in a career or job description. Sometimes you have to carve out of every opportunity you have what you love about it and make the most of that because you love it so much and eventually you create your dream job.

No risk, no reward

I'll start this one by admitting there are plenty of people who are rewarded financially without taking much risk at all. The 'investors' who make it big with OPM (other people's money), the trust fund kids, the baby boomers who rode a real-estate wave and retired wealthy almost regardless of what they did, etc. Financial reward is not the kind of reward I'm addressing here. I'm not giving financial advice here; I'm giving heartfelt career advice. To have a rewarding career, one must take risk. That risk doesn't always have to look like the big gamble - like mortgaging your house to finance a startup. Sometimes its about trade-offs or sacrifices or taking the path few people agree is the right one.

Again, this comes back to following your passion and following your instincts. One of my favourite jobs was teaching. When I decided to take my Bachelor's of Education there were no teaching jobs - no one thought it was a smart career choice. I didn't know better, I just chose to ignore the common wisdom and follow my instincts - follow my interests, pursue my passion for learning (for which all things related to 'school' was a surrogate). I took that risk. A great teaching job was my reward.

In 2006 (later in life), I left a great job with great pay with a great company with great growth potential in a great industry with a great mentor to start my own consulting company. No one thought that was a good idea. I took that risk. My reward was six years of the kind of education which only exposure to an enormous breadth and depth of companies, technologies, people, methodologies, and business models can provide.

Sometimes the risk you need is not making a change. For me, the risk of not exploring 'the alternative' is one of the hardest to accept. For the last few years, taking the risk of staying put and not chasing the next shiny opportunity has rewarded me with the lessons I needed to learn by seeing things through.

There are others who consider the reward to be all about minimizing all avoidable risk. "No risk is the reward" is their mantra but they are risking something - maybe it is living a more interesting life or seeking out the rewards of a life that takes risk

There is always a 'risk'. Whether you think so or not, every time you choose to do something you're inevitably deciding not to something else. Be conscience of that. Be okay with that. Be aware of the rewards of your decision - even ones you hadn't anticipated or were originally the basis of your decision - but also be aware to what you're not doing by putting yourself on a particular trajectory.

All that leads me to my own bonus 'platitude': trust your instincts as much as your logic. I wish my friend John had let himself be a professional musician because he would have rocked it but that wasn't his path and I believe he has been true to himself. I've been told so many times I should 'be' various things I was good at that were not my passion. Many of these things were very logical - clear job opportunities with lucrative and secure futures - but they did not align with my passion. I've also left things I was very much enjoying and passionate about to pursue things I was even more passionate about.

You'll face those moments where you feel pressured to make the 'right' choice - by family or friends or advisors or even the logical side or your brain. Don't ignore that but - and this is particularly important for those of you who love math/science - logic/reason is much more contextual than most care to admit. Logic - in the philosophical sense - would not have you ignore your passions, your interests, your loves. The earlier you learn in life (and in your career) to follow what some might call your 'instincts', the happier you will be.

So what has this lesson been about? Firstly, know yourself and prepare to be true to what drives you. Sometimes your job will be a means to affording your passion, sometimes your passion will decide your career, and sometimes you'll find - or even create - jobs which suit your passion.

People often say "find your passion" as if it were some treasure which, when found, is the magic to happiness. I prefer - as the image says above - "live your passion" because it is a journey and you know what they say about journeys vs destinations.

Make choices to do what you love and love what you do and when you do you'll be good at it and that - quite simply - is the reward.

6 Lessons for Mentees from a Seasoned Career-Changer (a series)

noreply@blogger.com (Lee Buckler) — Fri, 19 Nov 2021 16:12:00 +0000

I've had wonderful mentors throughout my career. Recently I've had several invitations and opportunities to start paying back by being a mentor at work, through the ESP program sponsored by ISCT, and giving talks to young professionals. It has really made me think about my own career and what advice I would give myself 30 years ago.

As part of that exercise I've come back to this blog to write down some of the hard-earned, experienced-based lessons I take with me into the last decade (or so) of my career.

I've broken down my thoughts into "6 lessons" - a draft outline sketched out below - which I will write about over the coming weeks. It's a useful exercise for me as I consider what value I can share my mentees and since I'm doing it anyway, I might as well share it in hopes that it triggers others to use, engage with, and share what they find useful or things they might add.

Lesson 1 - the platitudes

Follow your passions
Do what you love, and you'll be good at what you do
No risk, no reward

Lesson 2 - more platitudes and a kick in the pants

All good things come to those who wait (but not for those who wait around)
Be careful what you wish for you just might get it (so be prepared - go in eyes wide open)
I can't. I don't have time. I'm not qualified. I don't know how. And other lame excuses

Lesson 3 - its all up to you

It's your career. Manage it like a company
Perception is everything (or, at least, it is your reality)
Not deciding is the same as deciding (there's a time for thinking and a time for doing)
Pretending you know, helps no one. Be smart - look dumb - ask questions.

Lesson 4- its about the peeps

It's all about the people (invest in relationships)
Do unto others as you would have them do unto you
Never avoid conflict (but rarely engage in it - make the tough conversations the easiest ones)
Take time to be a mentor

Lesson 5 - be judged by how you helped

Make your mark by contributing (not boasting or boosting)
See a need - get on with (addressing) it
Small scale can scale so don't let the end scare you into not beginning
You can turn anything around, one person/thing at a time

Lesson 6 - thinking matters, doing matters more, thinking about what you're doing matters most

Goals are good, plans seldom work
Vision is good, execution is better
Never forget to spend some time looking at the big picture
Invest in your context

First allogeneic stem cell therapy approval in Japan

noreply@blogger.com (Lee Buckler) — Wed, 17 Nov 2021 18:36:00 +0000

In September 2021 Takeda Pharmaceutical's Alofisel (approved in the EU in 2018) was approved for the treatment of complex perianal fistulas in patients with non-active or mildly active luminal Crohn’s disease.

https://www.takeda.com/newsroom/newsreleases/2021/takeda-receives-approval-to-manufacture-and-market-alofisel-darvadstrocel-in-japan-for-treatment-of-complex-perianal-fistulas-in-patients-with-non-active-or-mildly-active-luminal-crohns-disease/

This was Japan's first allogeneic stem cell therapy approval.

Today, Japan’s reimbursement policy panel approved the listing of Alofisel, with an NHI price of 5.62 million yen (~USD $50,000). It will join the NHI price list on Nov 25. https://pj.jiho.jp/article/245593

Alofisel, granted RMAT designation in 2019, has not yet been approved in the U.S.

Foreign company pursuit of cell therapy commercialization in Japan: strategies and considerations

noreply@blogger.com (Lee Buckler) — Fri, 06 Aug 2021 17:13:00 +0000

I'm thrilled to announce that I will be presenting at the upcoming 5th Annual Cell & Gene Therapy World Asia, live streaming on 15th and 16th September 2021.

I will be presenting on "Foreign company pursuit of cell therapy commercialization in Japan: strategies and considerations" at 9:30AM (Singapore Time) on 16th September.

My presentation will cover the following topics:

· Unique constraints and opportunity for regenerative medicine in Japan

· Practical strategies for building a virtual team to support real progress

·       PMDA conditional approval -- not the only expedited commercialization pathway in Japan

In preparing for this presentation, I had opportunity to speak with Gladys Koh of IMAPAC to discuss

1.    key trends shaping the cell therapy industry moving forward in a post-pandemic world in Asia
2.    exciting technologies or developments that have caught my attention in the past year
3.    thoughts on how regulations pertaining to cell therapies can be evolved to allow the progress of the industry
4.    current struggles with respect to commercialization of cell therapies and how would they might be overcome
5.    key challenges this industry faces and is currently trying to overcome
6.    developments should the industry look out for from RepliCel Life Sciences Inc. in the coming year
8.    some of the key takeaways the audience can expect from my presentation

To view the interview, https://youtu.be/p2oZPbgFkMQ

Click here and here for a written advance peak at a few thoughts from some of the following speakers involved in the event such as:

Gregory Fiore, President & CEO, Exacis Biotherapeutics, USA

Zhimei Du, Global Head Process Cell Sciences, Cell/Gene Therapy, Merck & Co, USA

William Hung, Assistant General Manager, MariaVon, Taiwan

Hardy TS Kagimoto, Chairman & CEO, Healios, Japan

Pascal Touchon, President & CEO, Atara Biotherapeutics, USA

Joy (Shuxia) Zhou, Head of Drug Product Development, Cell Therapy, Takeda, USA

Magali Taiel, Chief Medical Officer, GenSight Biologics, France

Andreas Weiler, Global Head of External Supply Cell & Gene Therapy, Novartis, Switzerland

Seong-Wook Lee, President & CEO, Rznomics Inc., Korea

Yu Zhang, SVP & Chief Scientific Officer, VCANBIO Cell & Gene Engineering, China

Shi-Jiang (John) Lu, President and CEO, HebeCell Corporation

Devyn Smith, CEO, Arbor Biotechnologies, USA

Lee Buckler, President & CEO, RepliCel Life Sciences Inc.

Cell and gene data innovation and multi-stakeholder data transfer

noreply@blogger.com (Lee Buckler) — Thu, 15 Jul 2021 23:07:00 +0000

Hello readers. It's been awhile. I'm pleased to be working with Reuters on some events including this upcoming webinar on how to utilize RWD in cell and gene therapies with speakers from Novartis, BMS and Salesforce. Click here for more info.

The requirements of complex and delicate value chains within cell and gene therapies (CGT) is something pharma has traditionally never seen before. Leaders in CGT are innovating data sharing approaches by utilizing RWE and RWD across the value chain, and achieving multi-stakeholder alignment, to ensure commercial success.

Join C&GT delivery trailblazers BMS, Novartis and Salesforce to learn how to turn data in to true insights: that enhance the science, reduce the cost of deliver, improve commercial decision-making and elevate the patient experience.

Sign up for the webinar “Cell and gene data innovation and multi-stakeholder data transfer” here.

When you join, you will learn how to:

• Reimagine the technology: digitally transform existing data integration models for cell and gene therapies

• Reach for the future together: develop a multi-stakeholder strategy collaboratively, that aligns on needs and capabilities

• Cross-capture and utilize data flow from R&D through to launch to win the marketplace with science and data

• Prepare for and address the major barriers to success: such as infrastructure non-readiness, digitally siloed stakeholders, underdeveloped partnerships, and data illiteracy

• Turn data in to true insights: that enhance the science, reduce the cost of deliver, improve commercial decision making and elevate the patient experience

Tune in live to the free webinar on July 29 at 11am ET/4pm BST, or sign up to receive the recording here.

Please also feel free to share with your colleagues working in C&G therapy, so they can benefit too.

Lee

Will 2017 be the year of Cell Therapies?

noreply@blogger.com (Lee Buckler) — Tue, 24 Jan 2017 21:52:00 +0000

Here is the intro to a piece I published earlier this week on the Huffington post (click here for the whole article)

While 2016 may not go down as a lot of people’s favorite year, one development most people will agree to put a positive check mark beside is the near-unanimous passing of the 21st Century Cures Act in the United States.

In the last breaths of its tenure, the Obama Administration was able to bring both sides of Congress, the patient community and industry lobbyists together in a consensus on what was needed to drive better, faster medical innovation in the years to come. Faster cures to future patients.

There are a lot of reasons to be pleased with the many provisions of the 21st Century Cures Act but as the CEO of a cell therapy company, RepliCel Life Sciences Inc., I’ll focus this brief post on the area in which I’ve dedicated most of my professional life.

Out of the spotlight of the more prominent provisions of the Act, is a section pertaining to what I predict will be a growing but eventually radical boost to the development of regenerative medicines such as “cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products”.

Regenerative medicine is the idea that we can use cells or tissue from a patient or donor and do something more therapeutically significant for patients suffering from disease or conditions than we can do with drugs or other therapies. This next pillar of medicine dares to cure not just slow disease progression or reduce symptoms.

The 21st Century Cures Act promises not only foster the development of such therapeutic products but presents, I believe, exciting reasons to re-look at investing in cell therapy and other regenerative medicine companies.

Here are four such reasons:

Click here for the remaining article on Huffington Post

An Interview with RegMedNet. Bringing value to the online regenerative medicine community.

noreply@blogger.com (Lee Buckler) — Wed, 06 May 2015 15:16:00 +0000

As anyone who has followed my activity over any period of time will know, I'm a big fan of the power of social media to power business and careers if not entire industries, trends, movements etc. I'm a participant (some more, some less) in many different platforms for different purposes (some personal, some business). Consequently when a new online network comes across my radar it piques my interest.

Recently I had the pleasure of sitting down with Alexandra Thompson who is the Webinar & Community Manager for future science group in the UK to talk about the new online social network they are building around regenerative medicine.

Describe RegMedNet to us.

RegMedNet is a social network designed especially for the regenerative medicine community, aiming to help them showcase their news, views and insights to the rest of the community, and easily access those of others. By talking to authors and editorial board members of our journal and members of the regenerative medicine field at conferences, we realised there is not really one place where the whole community, including all disciplines and nationalities, could easily connect and communicate: we wanted to design a site that would act like an online, international, multidisciplinary conference, where people can showcase the latest news, share insights, debate hot topics, make connections and learn – but at any time and from any place.

What is lacking in other online activities or forums that you hope RegMedNet will address?

There are a lot of existing blog sites, websites and social media sites out there already, but with RegMedNet we have done three things. First, we have made it extremely easy to join and use, knowing time is a constraint for many in the community. Sign-up is free and has no lengthy forms or processes, and posting your own content and following others requires only a couple of clicks. Second, we have all the best features of various other sites together in this one network; blog posts, video posts, video chat, following, etc. Lastly, we have structured the site to cut out the noise you can get with social networks, making it easy for you to get the precise content you are interested in. You only get email alerts about people you choose to follow, but you can still visit anyone’s profile and view their content. Also, the site is organised into different ‘channels’ of interest such as ‘Research’ and ‘Industry developments’, so it is easy to find content relevant to you.

How does RegMedNet interact with people’s existing social media activity?

The idea for RegMedNet is that often people have more to say than say a Tweet but less than a published paper, which can also take a fair amount of time from submission to publication and therefore sharing! A lot of people have blogs already, and you can post those on RegMedNet or link over to these existing blogs, so RegMedNet becomes the hub of knowledge for all things regenerative medicine. You can also easily share content from RegMedNet to Facebook, LinkedIn, Google+ and Twitter, so you still get positive feedback into your social media streams.

Are you looking at using RegMedNet to supply additional interactive media content that supplement published papers in the journal?

We certainly are. A disadvantage with the traditional publishing model is the lack of showcasing of large numbers of video or image files. You can not only share these on RegMedNet, but also encourage further discussion of articles. You could show video tours of your lab or institution discussed in an institutional profile, a conference talk to supplement your conference report or a video of a protocol discussed in your research article. The possibilities are endless!

For those new to your site, how would you propose they start?

By signing up of course! Followed by completing their profile to showcase to the rest of the community who they are and what they know. There are various how-to guides in the RegMedNet channel to help users, but I hope that everyone will find the site fairly intuitive as it is designed with ease and simplicity in mind. If members are entirely new to blogging and vlogging, so am I! It is just about sharing what you know or think; as long as you are being respectful to others you can approach both however you like. Of course, as the Community Manager I will always be happy to help by offering advice and answering any questions.

What are you hoping people will say about RegMedNet and how it has helped them?

We have designed the site because we truly believe in the power of regenerative medicine. It is not only a priority investment for the future of healthcare, but offers hope to indications with no cure such as Huntingdon’s disease. Therefore, if people say that RegMedNet is a useful tool helping them to learn, connect and work together, which should help move the field forward more quickly and effectively, we would be delighted. I hope that down the line people say that it has helped international collaboration and standardization, as well as the unification of different disciplines encompassed by regenerative medicine.

What would be a successful first year look like for RegMedNet?

Community sites can take a long time to set up, so if we have a steady number of people signing up and then using the site over the next year we will be happy. By RegMedNet’s first birthday we are consistently getting positive feedback about the network, hearing that it is a useful tool that is helping its members to connect and collaborate etc. as hoped, I will be thrilled.

How to Grow a Regenerative Medicine Industry in Canada: Michael May of CCRM

noreply@blogger.com (Lee Buckler) — Fri, 24 Apr 2015 10:30:00 +0000

Under agreement with StreetWise Reports, I'm pleased to share their recent coverage of the regenerative medicine sector.

Original Source: Source: George S. Mack of The Life Sciences Report (4/24/15)
"How to Grow a Regenerative Medicine Industry in Canada: Michael May of CCRM"

There's a new kind of incubator in town. The Centre for Commercialization of Regenerative Medicine (CCRM) is a Canadian nonprofit that fosters hands-on association between academia, government, industry and investors to grow stem cell and regenerative medicine companies from the ground up. In this interview with The Life Sciences Report, CCRM President and CEO Michael May guides us through the process that brings ideas out of academia and adds the essential nurturing elements to get startups off the ground and into commercial development. Along the way, he mentions a few names that may interest investors.

The Life Sciences Report: You are the president and CEO of Centre for Commercialization of Regenerative Medicine (CCRM), a Canadian not-for-profit organization supporting development of technologies that will hopefully hasten the journey of stem cell and related therapeutics through regulatory pathways and to the market. How does it work?

Michael May: CCRM was founded through a unique government program called the Networks of Centres of Excellence. It was built around the concept of creating the right kind of ecosystem and network to drive commercialization. The government of Canada invests in three types of networks: academic; commercialization, which is where CCRM fits in; and what is called business-led networks—consortia of established companies that work together in an open innovation model.

CCRM actually followed on an academic network that was around for 14 years. That network had built a very strong culture of collaboration among the stem cell and regenerative medicine scientists in Canada, and prepared them to commercialize their technologies. CCRM was established to integrate elements over and above the discovery phase—access to industry, company creation and access to investors. And now CCRM has built an industry consortium of more than 40 companies from around the world. You could almost envision CCRM spinning off a business-led network one day to take full advantage of the government's strategy for building the right kind of ecosystem for commercialization.

TLSR: Does CCRM recruit innovators to Canada?

MM: Our model is to build a global network and to be a not-for-profit, neutral, safe place to bring together the right resources, elements and leaders, including innovators, to drive technologies forward. We, of course, need to look at a benefit for Canada, but we don't see a benefit to Canada as being mutually exclusive from the benefit to any investor or innovator or stakeholder around the world.

TLSR: How does CCRM differ from other industry advocacy organizations—for instance, from the Alliance for Regenerative Medicine (ARM), which was formed in the U.S. in 2009. I recently spoke with ARM Chairman Edward Lanphier about ARM. Are you familiar with that organization?

MM: Indeed I am. I'm on the executive committee for ARM.

CCRM is not just an advocacy group. It's actually built around tangible facilities and infrastructure that can drive not only technology development for out-licensing to our industry partners, but can also enable company creation in a very different way than has been done here in the past.

We have a tie-in to a network of academics and in-house facilities that develop technologies the way a startup company would. With our founding academic network of Canadian institutions and scientists, we have access to global innovators. We have been able to negotiate access to their intellectual property (IP). When we find IP, and after doing due diligence on it and bundling it with other IP, we can bring that technology into our own facility, where we have a staff of 35 people, including about 20 scientists, who can conduct wet diligence and product development. We fill a gap—we are doing work that academics typically don't like to do or don't always do very well. By bringing together all these capabilities, along with some specialized platforms, like cellular reprogramming, gene editing, cell manufacturing and biomaterial synthesis, we can complement what a standard academic network would bring to the table.

We are also driven by a government mandate to be sustainable, so when we invest in promising technologies or bundles of technologies, we expect to take some equity, or some position, in those opportunities. That way, over time, we can grow and sustain an industry around our network.

TLSR: Regenerative medicine appears to have been left behind in terms of development, compared to other biotechnologies. Why do you think these powerful breakthrough technologies have languished on the lab bench?

MM: The bottom line is that regenerative medicine is complicated. It's an expensive process to take on from start to finish.

Our model at CCRM is built around a couple of assumptions. One is that you have to do more with less these days, and so you need capital efficiency, especially at the very early stages, where investors really have abandoned projects. Although today they say there's a lot of investment in early-stage development, that typically means venture capitalists providing follow-on investment to their current companies. New startups are still having trouble getting financed. Regenerative medicine is also a new industry, so I think this "left-behind" feeling is symptomatic of that fact.

TLSR: You just used an important term: capital efficiency. How do you get around, or solve, this problem of doing more with less?

MM: I think it can be solved through collaborative vehicles. CCRM started with an academic network that was building collaboration and getting access to IP. We then leveraged the reputation of our academic network to attract industry. Then, of course, industry brought its ever-important validation and marketing savvy to the table, which represents expertise that is largely absent in the academic environment.

After three years of operation, with significant deal flow and two networks in place, CCRM is now at a stage where it's building the third key element of its model: an investor network. The system was primed with a very modest amount of money, but now we want to fuel our model with risk capital. Global networks, access to deal flow, sufficient funding and coordination of specialized infrastructure/expertise are all key elements of CCRM’s model for filling the gap between discovery and the market.

Investors are looking for good investments. If we can get companies over some of the early hurdles with unique resources, I think we will demonstrate accelerated commercialization.

TLSR: Michael, is it hard to get investors to invest in science projects? They really need to see the path to commercialization, don't they?

MM: Absolutely. Financiers don't like to take scientific risks. They are more comfortable with commercial risk. Despite what people think, scientists are also risk takers, but they don't like to take financial risk. The role of an entrepreneur is to sit in the middle and manage everyone's comfort or discomfort with different risks. That's an important role for an organization like CCRM and the entrepreneurs that it brings to bear.

TLSR: You have said that Japan and Canada would lead the way in regenerative medicine. Does that imply that the U.S. has lagged?

MM: I would never discount the ability of the U.S. to muster resources and drive innovation in any field. There are strengths in regenerative medicine across the U.S., but regenerative medicine has a unique status in Canada. Stem cells were actually discovered in Canada prior to the bone marrow transplants of the 1960s. Of course, Japan's Shinya Yamanaka received the 2012 Nobel Prize in Physiology or Medicine for discovering that mature cells could be reprogrammed to become "pluripotent." There is a nationalistic element to regenerative medicine in both Canada and Japan. That's one difference.

Japan has taken it one step further. It has taken a novel approach in its regulatory policy by acknowledging that cell-based products may require a different regulatory pathway than drugs or devices. In November 2013, the Japanese Diet (parliament) approved new legislation, the Regenerative Medicine Law, to provide for conditional approvals of cell-based products that demonstrate very strong safety data. These products can go to the market and be sold. Once enough patients have been treated to demonstrate proof of efficacy, then they get full approval. This is a very different model. In fact, if you think about it, if a company can sell its product after Phase 2 development with proof of safety, it changes the return on investment (ROI) model entirely for early-stage programs.

Canada has been the first to approve regenerative medicine products. It was the first country to approve the early skin substitutes from Organogenesis (private) and Advanced Tissue Sciences (Chapter 11 liquidation under bankruptcy in March 2009). Canada was also the first to approve Osiris Therapeutics Inc.'s (OSIR:NASDAQ)(OSIR:NASDAQ) Prochymal (remestemcel-L). Where Canada needs to be innovative now is with reimbursement. None of those products has been reimbursed in Canada.

TLSR: Is CCRM working with regulators on reimbursement?

MM: Yes, it's one of the areas of focus for CCRM. Who is going to pay for a therapy is typically the last question people ask in the development process. We are working with government agencies in Canada so that the reimbursement issue gets addressed early in development. That would put Japan and Canada at the leading edge of innovation in the commercialization of these products.

TLSR: You have referenced Japan's new regulatory legislation. Does Canada propose to do anything like that, so that companies could effectively get a drug on the market with strong Phase 2 safety data?

MM: Canada is not proposing legislation like that—at least that I'm aware of. What the Canadian regulatory authority, Health Canada, has demonstrated in the past is that it will approve cell-based products on a conditional basis, just like Japan. Health Canada is focusing more on risk/benefit, and is therefore flexible. It has also demonstrated that it will approve cell-based products much faster than other jurisdictions. So although Canada doesn't appear to be going down the same regulatory route as Japan in creating a formal Phase 2 approval policy, it is being very clear about its willingness to be open-minded and to be both a cost-and time-effective regulator of these new therapies.

TLSR: Risk/benefit implies efficacy, not just safety.

MM: It does. It also implies that if patients have no other option, then having an experimental therapy available quickly fits into the risk/benefit equation. That's part of the flexibility embedded in the Canadian system. About six months ago Health Canada produced a guidance document for cell-based clinical trials to try to provide not only flexibility in its regulation but also some clarity on what it's expecting in terms of clinical data.

TLSR: Tell me about some of the interesting companies developing regenerative medicine technologies in Canada?

MM: One of our leading company creations (private) concerns the expansion of hematopoietic stem cells from cord blood. Getting the levels of stem cells to treat leukemias in adults, versus children, has been a clinical development goal for some time. We have brought together a couple of technologies from different institutions, along with some bioreactor technology from one of our industry consortium partners, to deliver best-in-class hematopoietic stem cell expansion, gene therapy and potentially the ability to produce mature blood products.

Another area where Canadian researchers have been leading is in cancer stem cells and the thesis that cancer is actually driven by a stem cell that's gone awry. If you can find and target that cancer stem cell, you can target the cancer. One of the companies that CCRM has funded in the past year is Actium Research (private), which is raising money now. Its technology is based on the work of Dr. Mick Bhatia, a senior scientist at McMaster University in Hamilton, Ontario. Actium has created a unique platform to screen molecules that will attack cancer stem cells as opposed to normal stem cells.

TLSR: Is Actium's technology about identifying cell membrane antigens that might be exclusive to a cancer stem cell?

MM: That could be part of it. The company is identifying ways to attack a cancer stem cell versus a normal cell. For instance, if you can push a stem cell to differentiate, you can kill it with standard treatments. Because a stem cell is in a pluripotent state, it's more resistant to drug therapy. There are a number of different mechanisms there, but Actium is a good example of a very young Canadian company that is focused on the country's strength in stem cell research.

TLSR: What about another name? Do you have a public name or two that investors could access?

MM: Yes. RepliCel Life Sciences Inc. (RP:TSX.V; REPCF:OTCQB) is focused on cell therapy for orthopedic and hair restoration applications. It too is a Canadian company, and it has been aggressively developing ties to Japan, in association with its partnership with Shiseido Company Ltd. (4911:TSE), around transplanting cells for hair growth. The company has a diversified portfolio of technologies, is very focused and is growing rapidly as a public company.

TLSR: Many cell therapy companies have not participated in this tremendous biotech bull market of the past few years. RepliCel's stock has done quite well over the past 12 weeks, while most cell therapy companies have not. Do you have a thought on why that might be?

MM: I think, in part, that comes down to the leadership of the company and its understanding of what it takes to not only advance a technology, but to also tell its story. RepliCel has made some key hires in the last year to augment and strengthen its offering. The company has experienced management, and we all know that's a key piece of the puzzle in moving these companies forward.

TLSR: Is there another public company you might mention?

MM: Canadian company Sernova Corp. (SVA:TSX.V) is an interesting name. It has developed a unique vascularized pouch for cells, and it is working in the same area as San Diego-based ViaCyte Inc. (private) in the U.S., which has been funded by the California Institute for Regenerative Medicine. Sernova is working with Dr. James Shapiro, at the University of Alberta in Edmonton, around transplanting islets, or islet-like cells, to treat diabetes. Sernova is focused on the device to transplant those cells.

TLSR: Thanks for your insights, Michael.

Michael May is president and chief executive officer of the Centre for the Commercialization of Regenerative Medicine (CCRM). Prior to CCRM, he was president, chief operating officer and cofounder of Rimon Therapeutics Ltd., a Toronto-based regenerative medicine company developing novel medical polymers that possess druglike activity. May completed his Ph.D. in chemical engineering at the University of Toronto in 1998 as a NSERC (Natural Sciences and Engineering Research Council of Canada) Scholar, and was awarded the Martin Walmsley Fellowship for Technological Entrepreneurship. May sits on a number of boards and advisory committees, including MaRS Innovation, the Alliance for Regenerative Medicine, 20/20 Vision and the Department of Chemical Engineering and Applied Chemistry at the University of Toronto.

Want to read more Life Sciences Report interviews like this? Sign up for our free e-newsletter, and you'll learn when new articles have been published. To see a list of recent interviews with industry analysts and commentators, visit our Interviews page.

DISCLOSURE:

1) George S. Mack conducted this interview for Streetwise Reports LLC, publisher ofThe Gold Report, The Energy Report, The Life Sciences Report and The Mining Report, and he provides services to Streetwise Reports as an independent contractor. He owns, or his family owns, shares of the following companies mentioned in this interview: None.

2) The following companies mentioned in the interview are sponsors of Streetwise Reports: RepliCel Life Sciences Inc. The companies mentioned in this interview were not involved in any aspect of the interview preparation or post-interview editing so the expert could speak independently about the sector. Streetwise Reports does not accept stock in exchange for its services.

3) Michael May: I own, or my family owns, shares of the following companies mentioned in this interview: None. I personally am, or my family is, paid by the following companies mentioned in this interview: None. Senova Corp. and RepliCel Life Sciences Inc. are fee-paying members of CCRM industry consortium. CCRM is a shareholder of Actium Research. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.

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Select 2015 Stem Cell Company Catalysts: Sagient Research's Edward Stopke

noreply@blogger.com (Lee Buckler) — Fri, 23 Jan 2015 01:23:00 +0000

Under agreement with StreetWise Reports, I'm pleased to share their recent coverage of the regenerative medicine sector.

Original Source: Source: Peter Byrne of The Life Sciences Report (1/22/15)
"Convert Catalysts into Profits: Sagient Research's Edward Stopke"

The biotech sector is teeming with companies racing to bring the hot new drug or therapy to the marketplace. But realistically assessing the therapeutic potential of pipeline products is the healthy approach for selecting a winner. In this interview with The Life Sciences Report, Edward Stopke of Sagient Research unveils his list of companies with catalytic moments in the making.

The Life Sciences Report: What types of catalysts affect biotech stocks?

Edward Stopke: The primary catalyst affecting a biotech stock is trial data, especially large Phase 3 trials that test the compound against a comparator. The other main catalysts emerge from regulatory interactions with the U.S. Food and Drug Administration (FDA) or the European Medicines Agency. Product safety rulings can drastically affect the trajectory of a biotech stock, of course. But investors need to watch a company's interactions in meetings with the advisory committees serving the governmental agencies. These interactions can presage whether or not the agency will eventually approve the drug.

TLSR: Are there economic catalysts that affect the life sciences sector as a whole?

ES: Pending changes in pricing policies for therapies can catalyze shareholder actions. Historically, many companies have been able to price high without too much backlash from payers. Companies have typically charged less for treatments with large patient populations, and the more expensive treatments target smaller numbers of patients. But during the last year, some high pricing became less sustainable. I am thinking of Gilead Sciences Inc.'s (GILD:NASDAQ) pricing for Sovaldi, its treatment for hepatitis C virus (HCV), which caused quite a stir with insurers and governments alike. Companies generally argue that high prices are in line with previous treatments and that the products offer superior efficacy. But the sheer number of eligible patients for a high-priced drug can strain entire healthcare systems. I predict that during 2015 payers will force prices downward, not just for highly prevalent diseases such as HCV, but in all therapeutic markets, stem cells included.

TLSR: What catalysts are coming up for stem cell companies in 2015?

ES: Cytori Therapeutics Inc. (CYTX:NASDAQ) is developing a stem cell therapy from fat-derived stem and regenerative cells. It has Phase 1/2 data for heart disease slated for release in Q1/15. Vericel Corp. (VCEL:NASDAQ), which recently changed its name from Aastrom Biosciences Inc., has Phase 2 data expected in H1/15 on tissue repair cells for heart failure. Athersys Inc.
(ATHX:NASDAQ) is developing a stem cell product called MultiStem.

TLSR: What are the projected applications for MultiStem?

ES: Athersys is studying MultiStem in human trials for stroke, myocardial infarction (MI) and graft-versus-host disease (GvHD). It is in Phase 2 development for stroke and early Phase 1 for MI and GvHD. The firm completed enrollment in its Phase 2 stroke study before the new year, so the first 90-day results will emerge in Q1/15E. Athersys plans to progress its MI program into Phase 2 in Q1/15. The company is looking for a development partner to further its program. Announcing a partnership deal would signal investors that experts have faith in MultiStem, and Athersys' stock price would respond positively.

TLSR: Who else do you like in the stem cell space?

ES: bluebird bio Inc. (BLUE:NASDAQ) is developing its Lenti-D product, which is more of a gene therapy product, but does consist of the patient's own stem cells. These are modified by the lentiviral vector to deliver genetic material into the cells themselves. There is a lot of hype floating around this company. It is currently in Phase 2/3 studies for a rare genetic disease, adrenomyeloneuropathy, which is similar to multiple sclerosis. Hopefully, we will see data for that pipeline product next year.

TLSR: What other therapeutic spaces do you like for strong catalysts in early 2015?

ES: Neuralstem Inc. (CUR:NYSE.MKT) has a major depressive disorder product in clinical trials code named NSI-189. It is an oral compound designed to stimulate neurogenesis of the hippocampus, which could potentially reverse the atrophy seen in depression and schizophrenia. So far, we have only seen earlier preclinical and Phase 1 data, but those results have shown meaningful reductions in both cognitive and depressive symptoms in patients who are on active therapy. And the treatment has been well tolerated.

TLSR: How does Neuralstem's product differ from competitive products?

ES: There are many treatments on the market for treating depression, of course. Most of these products, however, are small molecules. They work by inhibiting the reuptake of a combination of norepinephrine, serotonin and/or dopamine. Neuralstem's treatment uses the patient's own neural stem cells to protect against damage to the nervous system itself, and to repair existing damage, too.
TLSR: What expertise do Neuralstem's managers bring to the marketplace?

ES: Neuralstem's chief scientific officer and senior vice president of research previously worked at the National Institutes of Health's Laboratory of Molecular Biology, where they researched the isolation of human neural stem cells.

TLSR: Do you see any other potential catalysts for Neuralstem?

ES: If all goes well, Neuralstem's NSI-189 product will move into Phase 2 development in Q2/15, so that is a good advancement opportunity. We should see topline results in the early part of this year for the firm's other stem cell products, including NSI-566. That treatment is currently being studied in a Phase 2 trial for Lou Gehrig's disease.

TLSR: Who is making waves in the cancer treatment space?

ES: There is a lot of excitement surrounding the chimeric antigen receptor (CAR) T-cell immunotherapies that engineer the patient's own immune cells to target tumor-specific molecules. Kite Pharma (KITE:NASDAQ) is developing its CAR T programs for hematologic cancers, and its KTE-C19 program has shown high response rates in earlier Phase 1/2 studies. Kite just bought a licensing deal with Amgen Inc. (AMGN:NASDAQ). The deal is related to the next generation of immunotherapies based on Kite's cell therapy platform. Celgene Corp. (CELG:NASDAQ), Novartis AG (NVS:NYSE) and Juno Therapeutics (JUNO:NASDAQ) are working on similar programs.
Rexahn Pharmaceuticals Inc. (RNN:NYSE.MKT) has a couple of clinical-stage oncology candidates. Its most advanced compound is known as Archexin. It targets the PI3K pathway and is being studied in a Phase 2 trial for renal cell cancer. While we have not yet seen data yet from this compound, last year the FDA approved Gilead's Zydelig for lymphoma, which has a similar mechanism of action. Other large pharma companies, such as Novartis and Merck and Co. Inc. (MRK:NYSE), are studying similar compounds in various oncology indications. The research synergy with these big firms could prove fruitful for Rexahn in terms of acquisition potential.

TLSR: I see that Rexahn recently appointed Richard Rodgers to its board. What does Rodgers bring to the company?

ES: He brings experience with in- and out-licensing, as well as mergers and acquisitions. He was previously with Abraxis BioScience Inc. until it was acquired by Celgene. He was also with MGI Pharma Inc., which was acquired by Eisai Inc. (ESALF:OTCPK).

TLSR: What types of specific catalysts are likely to affect Rexahn's stock price?

ES: If Rexahn can harness Rodgers' experience to secure a licensing deal for Archexin, its stock price would obviously respond positively. It has a few other early-stage compounds in various tumor types. If any of these products survive later-stage studies, the market will provide rewards. We could see some Phase 1 data from Rexahn's RX-5902 and RX-3117 compounds in the near future. These are being studied for very solid tumor indications.

TLSR: What other biotechs with emerging catalysts do you follow?

ES: I like quite a few smaller companies, like Raptor Pharmaceutical Corp. (RPTP:NASDAQ), Celator Pharmaceuticals (CPXX:NASDAQ), Sarepta Therapeutics Inc. (SRPT:NASDAQ), Celldex Therapeutics Inc. (CLDX:NASDAQ), PTC Therapeutics Inc. (PTCT:NASDAQ) and Rockwell Medical Inc. (RMTI:NASDAQ). Each of these has a unique pipeline.

TLSR: Can you synopsize where each of these companies is at in the pipeline?

ES: Raptor Pharmaceutical's main compound is Procysbi. It is already on the market. It was approved in 2013 to treat cystinosis, and it is one of only three such treatments approved in the U.S. Raptor is also studying Huntington's disease and nonalcoholic fatty liver disease, the latter being of notable interest due to its large market size and connection to obesity. Raptor is expecting results from its larger Phase 2b study in H1/15.

Celator Pharmaceuticals is developing CPX-351 for leukemia. It began Phase 3 development in late 2012 with about 300 patients. The first results from this study are expected sometime in Q2/15.
Sarepta Therapeutics has a number of compounds. The most advanced is eteplirsen, developed for a type of muscular dystrophy. We have seen clinical data on it, and it is fairly good, although taken from a small subset. There is a lot of speculation about whether the FDA will accept Sarepta's new drug application (NDA) and what kind of data the agency will require for approval. Sarepta expects to file around the middle of this year. A successful filing and acceptance could move the stock price nicely.

PTC Therapeutics is developing its ataluren compound for muscular dystrophy. The company submitted a rolling NDA to the agency in late December. It is not yet a complete application, but it does allow completed portions of the application to be submitted and reviewed on an ongoing basis. PTC hopes to complete the application in late 2015. We should see the first results from its larger Phase 3 study toward year-end 2015.

Celldex Therapeutics' most advanced candidate is rindopepimut, which is being developed for glioblastoma, brain cancer. Topline results for a large Phase 3 are expected in the middle of this year. If the results prove positive, Celdex shareholders will benefit, as rindopepimut is the firm's main compound in a large treatment space.

Rockwell Medical has a single compound called Triferic. It is being reviewed by the FDA as a treatment for iron deficiency in chronic kidney disease patients. The agency is expected to give a decision on provability by the end of this month.

TLSR: Aside from handicapping the pipeline products, what qualities do you look for in a firm?
ES: It is vitally important to understand the capital structure of a company. As far as a company's potential staying power, I look at the general platform. Is it a stem cell platform? Is it a genetic therapy type of platform? Is it a completely new mechanism of action? Is it something that could be used in various types of diseases?

TLSR: Sounds good, Edward. Thank you for speaking with us.

ES: Thank you, Peter.

Edward Stopke is a financial analyst with BioMedTracker and has been with Sagient Research for three years. He is responsible for day-to-day analysis with the BioMedTracker analyst team, and works with the scientific analysts to determine the financial and market impact of early-stage drugs. In his time with Sagient, Stopke has gained an understanding of the pharmaceutical and biotech development process to better develop revenue models for various indications. Stopke received a bachelor's degree in economics from San Diego State University.

Want to read more Life Sciences Report interviews like this? Sign up for our free e-newsletter, and you'll learn when new articles have been published. To see a list of recent interviews with industry analysts and commentators, visit our Interviews page.

DISCLOSURE:
1) Peter Byrne conducted this interview for Streetwise Reports LLC, publisher of The Gold Report, The Energy Report, The Life Sciences Report and The Mining Report, and provides services to Streetwise Reports as an independent contractor. He or his family own shares of the following companies mentioned in this interview: None.
2) The following companies mentioned in the interview are sponsors of Streetwise Reports: Rexahn Pharmaceuticals Inc., Neuralstem Inc., Athersys Inc. The companies mentioned in this interview were not involved in any aspect of the interview preparation or post-interview editing so the expert could speak independently about the sector. Streetwise Reports does not accept stock in exchange for its services.
3) Edward Stopke: I own, or my family owns, shares of the following companies mentioned in this interview: Gilead Sciences Inc., Sarepta Therapeutics Inc. I personally am, or my family is, paid by the following companies mentioned in this interview: None. My company has a financial relationship with the following companies mentioned in this interview: None. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
4) Interviews are edited for clarity. Streetwise Reports does not make editorial comments or change experts' statements without their consent.
5) The interview does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer.
6) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their families are prohibited from making purchases and/or sales of those securities in the open market or otherwise during the up-to-four-week interval from the time of the interview until after it publishes.

Streetwise – The Life Sciences Report is Copyright © 2014 by Streetwise Reports LLC. All rights are reserved. Streetwise Reports LLC hereby grants an unrestricted license to use or disseminate this copyrighted material (i) only in whole (and always including this disclaimer), but (ii) never in part.

Streetwise Reports LLC does not guarantee the accuracy or thoroughness of the information reported.
Streetwise Reports LLC receives a fee from companies that are listed on the home page in the In This Issue section. Their sponsor pages may be considered advertising for the purposes of 18 U.S.C. 1734.
Participating companies provide the logos used in The Life Sciences Report. These logos are trademarks and are the property of the individual companies.

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Where Do Cures Reside? ARM Executives Think the Answer Is in Regenerative Medicine

noreply@blogger.com (Lee Buckler) — Fri, 09 Jan 2015 20:46:00 +0000

Under agreement with StreetWise Reports, I'm pleased to share their recent coverage of the regenerative medicine sector.

Original Source: George S. Mack of The Life Sciences Report (01/18/2015)
http://www.thelifesciencesreport.com/pub/na/where-do-cures-reside-morrie-ruffin-and-michael-werner-of-the-alliance-for-regenerative-medicine-think-the-answer-is-in-regenerative-medicine

Where Do Cures Reside? Morrie Ruffin and Michael Werner of the Alliance for Regenerative Medicine Think the Answer Is in Regenerative Medicine

The practice of medicine is being transformed. The journey has been arduous, but revolutionary stem cell, gene and immunocellular therapies are rapidly moving toward pivotal milestones—and investors in the space should strap in for a rewarding joy ride. In this interview with The Life Sciences Report, Alliance for Regenerative Medicine cofounders Morrie Ruffin and Michael Werner consider how new federal guidance might enable new standards of care in cardiovascular, neurologic and oncologic medicine, and offer a preview of next week's Biotech Showcase in San Francisco.

The Life Sciences Report: On Dec. 22, the stem cell and regenerative medicine space saw a very significant development. The U.S. Food and Drug Administration (FDA) published a new draft guidance paper on what constitutes minimal manipulation of human tissues and cells. Although this document is not binding, it does tell us what the agency is thinking. In effect, tissues and cells that have been processed in some way will be treated as drugs, and will come under the regulatory umbrella of the FDA. What does this mean for the industry?

Michael Werner: As sometimes happens with government agencies, major regulatory documents are released right before the holidays. As far as the Alliance for Regenerative Medicine (ARM) is concerned, our member companies are currently looking at the paper and trying to digest it. This draft guidance is open for comment for 60 days after its publication in the Federal Register. We will go through it with a fine-tooth comb, and we will prepare formal comments, which we will submit to the FDA early this year.

Generally speaking, I think the most significant aspect of the guidance is that it provides greater clarity on the FDA's definition of minimal manipulation and, therefore, which products can qualify as minimally manipulated, and which will be regulated as biologics or drugs.

What ARM has said, from its inception many years ago, is that this industry needs a clear and predictable regulatory pathway. The regulations for minimal manipulation have been around for a while, but as this field has expanded, and as the technology has changed, questions have arisen about how the FDA is applying its regulations, and what “minimally manipulated” means in the context of new, tissue-engineered products.

At the very least, it's important that the FDA has, through this document, been transparent about its views.

TLSR: Michael, will you explain further why this transparency is important?

MW: Companies need to know what the FDA is thinking. We've had conversations with individual companies that think the FDA will regulate them as a 361 HCT/P, but are not totally sure, which makes it hard to plan clinical programs. The fact that the FDA has come forward with a document like this is great. As always, the devil is in the details, and we will certainly be going over the document carefully. But published guidelines are a good thing.

TLSR: Could this guidance, in effect, run retail or storefront stem cell therapeutic clinics out of business?

MW: Time will tell. In terms of its impact on what you describe as storefront, I think it really comes down to this: If you are running one of those kinds of operations, or if you perform so-called stem cell tourism, you are now on notice about what kinds of technologies, processes and products are subject to FDA regulation. If you're a storefront clinic, you now know that the FDA not only believes it has jurisdiction to regulate, but how it's going to regulate in the days ahead. It lays the foundation for FDA's enforcement actions going forward.

I think we can expect the agency to use its enforcement discretion in a more consistent way. How aggressive it will be in terms of going after storefront clinics remains to be seen.

TLSR: Do you feel that this, in the long run, is going to be positive for the regenerative medicine and stem cell industry?

MW: I think it will. If you're a product developer, what you want is clarity. You want to know where the goalposts are, and you don't want the goalposts to keep moving. If the FDA is transparent, then that's 80% of the ballgame for the product developer.

Now, in terms of specific policies, the specific ways FDA is going to apply the regulations—that remains to be seen. Companies can—and will—go back to the FDA and say "Here are our thoughts about some of the specifics." Of course, each individual company must go through an iterative process with the agency regardless.

TLSR: It feels like the track to progress, and to the market, for the regenerative medicine industry has been even more difficult than it was for monoclonal antibodies, for lack of a better comparator. What do you think has held the industry back?

MW: One thing has been the clarity of the regulatory pathway. You describe the industry as being held back, but I'd like to say, more positively, that more clarity and predictability in the pathway would enable more products to move forward.

Another key issue involves standards, which has been acknowledged by the FDA and industry as important in terms of enabling more products to come to market. We also need changes to the reimbursement and payment systems, both public and private, to reward innovation. These are different products and models, differentiated by how they treat diseases at the underlying molecular and cellular level, and they could lead not only to treatments, but also to cures.

We need support from the U.S. government in terms of policies, in the same way that governments in Japan, the United Kingdom and elsewhere around the world have been enacting policies specifically designed to support the sector. The U.S. government needs to do the same.

The field is now poised to make a significant impact on healthcare. It's hard to say what's held the field back, but reimbursement is certainly a challenge that we need to get our hands around, to make sure the sector reaches its potential.

TLSR: Morrie, did you want to comment? The cell therapy/regenerative medicine sector has been in development now for about two decades-plus, yet I see only a couple of stem cell companies with a market cap above $1 billion ($1B), and all the rest are in the penny-stock to $500 million category. Why have cell technology companies lagged biotech?

Morrie Ruffin: We actually don't see it that way. That's one of the things we will show in the data that will be coming out in our Regenerative Medicine and Advanced Therapies State of the Industry Briefing on Jan. 12 at the Biotech Showcase in San Francisco.

Clearly, as in any sector, there are going to be successes and failures, but we do see a very significant and recognizable upward trend in the amount of money being raised in the sector. The cancer immunotherapy space is drawing a lot of interest right now.

But if you look across the board at cell therapy, gene therapy and immunocellular therapy companies, we count nine to 10 public companies with $1B-plus market caps in the sector right now. And there are obviously a number of private companies raising significant amounts from private investors as well.

TLSR: Morrie, are we on the verge of seeing pivotal data come out of companies in the sector?

MR: We are about to see a number of very significant events in the sector. Clearly, both those working in the industry and investors are looking for positive clinical events—milestones—that signal we are making progress in the clinic, and that we understand how we might commercialize these therapies. Over the next year or two, you will see a number of very significant data events around cell therapy. We are already seeing this in the oncology space, which is why there is so much interest in what's happening with cell-based or immunotherapies. This has led to a significant interest in cellular therapies across the board. Investors are going to hear a lot in the near future, as people begin to understand the clinical milestones that are going to drive this sector over the next 12–18 months.

TLSR: What do you think is the greatest obstacle to this industry moving forward? Is that issue being addressed currently?

MR: One of the challenges in this space—and I think this is to be expected in any evolving sector—is the issue of commercialization, which concerns manufacturing, scale-up and all the things required to make these new products available and successful. I believe that we have made tremendous progress just in the last few years in understanding what this will take, whether it's an allogeneic therapy or an autologous therapy. I think this is one of the reasons we see manufacturing and tool companies making significant investments in the sector. Companies like GE Healthcare (a unit of General Electric Co. [GE:NYSE]), Thermo Fisher Scientific Inc. (TMO:NYSE), the Lonza Group AG (LONN:SIX; LO3:FSE; LZAGF:OTCPK) and others are heavily engaged in this space. They see the huge opportunity here.

The cell therapy companies also understand that infrastructure is going to be an important part of how these therapies are brought to the market, and to the patient. One of the things we have been anticipating is having the processes of manufacturing, handling, and scale-up catch up with the lab bench science, and then duplicating that science on a large, profitable scale. I think we are on the cusp of that.

TLSR: Michael, you raised a point about government being involved in regenerative medicine, and you mentioned Japan, which has a new regulatory pathway in place whereby companies, in effect, have to show safety and the equivalent of Phase 2 proof of concept to get conditional approval for a cell therapy. Do you see the U.S. doing anything like that?

MW: I'll put it this way: I think it's possible the U.S. will take steps analogous to what other countries are doing to support the field. Is the FDA going to give conditional approval if a company shows certain kinds of safety data? I'm not sure about that, but I do think it is possible to take steps to smooth the pathway forward. For instance, we've talked with the FDA about standards in product development and manufacture. This is an idea that the FDA itself has said is critical to its product review process. Other ideas could include expedited approval programs. Such a program was recently created in the U.S. to support development of antibiotics.

The question is whether the kinds of programs that already exist can be applied or adapted to regenerative medicine, cell therapy and gene therapy products. I think you're more likely to see changes in existing U.S. policy, rather than copying Japan's policy or copying what other countries are doing.

TLSR: What do you think will be the motivating factor for policymakers to accelerate cell therapy development?

MW: There is more recognition that this sector could, potentially, cure diseases that have gone untreated or have been deemed incurable. In diseases like stroke or heart disease, we are basically treating symptoms. We are certainly helping people, but we are not actually curing disease. We're not getting people all the way back to health.

I think there's a greater recognition on the part of policymakers, as well as others, that regenerative medicine—and we use that term very broadly—is where cures and treatments are going to reside. Therefore, you're starting to see more acknowledgment of that. The U.S. government needs to make sure there are policies in place that will allow this industry to thrive.

TLSR: Morrie, you and Michael are going to be at the Biotech Showcase in San Francisco. What will you and ARM members be doing for attendees?

MR: One of the things we're doing, at the kick-off for the showcase at 8 a.m. on Monday, Jan. 12, is presenting our Regenerative Medicine and Advanced Therapies State of the Industry Briefing. This will be our fifth briefing at the Showcase. In the two-hour briefing, we will provide information on the performance of the sector. We will look back at 2014 and 2013, highlighting the progress made in the sector. Then we will look forward to what we anticipate to be major milestones and major events over the coming two years.

TLSR: Will there be an emphasis on any particular topic, or will it be a very broad briefing?

MR: We will be talking about several different things, but we plan to spend a lot of time talking about the progress being made in the gene therapy sector and genetically modified cell therapies.
When we talk about advanced therapies in regenerative medicine, we are being inclusive of all the companies working in the in vivo and ex vivo gene therapy areas, whether that's cancer immunotherapy, or CAR (chimeric antigen receptor) T-cell receptors, or other strategies being employed to harness the immune system to attack cancer. This will be a big part of the briefing. We will also look forward to a number of the major data events that we anticipate in 2015, in a number of indications, including stroke, cardiovascular disease and a number of neurodegenerative disorders.

TLSR: Thank you both for your insights.

Morrie Ruffin has more than 20 years of experience in the biotech and healthcare industries. He is a founder and managing director of the Alliance for Regenerative Medicine, the global organization representing the interests of the regenerative medicine community. Ruffin is also the managing partner of Adjuvant Partners, a boutique regenerative medicine and advanced therapies business consulting firm. Prior to joining Adjuvant Partners, he was the chief executive officer of LifeTech Innovations LLC (LTI), a business development consulting firm. Prior to his position at LTI, he was executive vice president of capital formation and business development at the Biotechnology Industry Organization (BIO), the largest trade organization representing the biotech and drug development industries. Prior to joining BIO, Ruffin worked for U.S. Senator Arlen Specter for five years as his senior legislative assistant. He received his master's degree in international studies and economics from the Johns Hopkins School for Advanced International Studies and his bachelor's degree from the University of Virginia.

Michael Werner is a partner in Holland & Knight's Washington, D.C. office. He has almost three decades of healthcare law, lobbying, regulatory, reimbursement and policy development experience in Washington. He is also is the cofounder and executive director of the Alliance for Regenerative Medicine, a Washington, D.C.-based organization whose mission is to advocate for federal funding, regulatory and reimbursement policies that will advance regenerative medicine research and product development. Before joining Holland & Knight, Werner was president of The Werner Group, a Washington, D.C.-based firm that provided lobbying, regulatory, and bioethics consulting services for biotechnology and pharmaceutical companies, physicians, health plans, investors, and patient advocacy groups. Prior to founding The Werner Group, he was chief of policy for the Biotechnology Industry Organization (BIO), representing over 1,000 biotechnology companies in the U.S. and other countries. Werner is also a founding member of the Board of Directors of the Coalition for the Advancement of Medical Research. He was senior healthcare advisor to U.S. Senate Majority Leader George Mitchell, a congressional investigator for the U.S. Senate Special Committee on Aging, and senior advisor to Maryland Governor William Donald Schaefer. Werner is a frequent media commentator and has appeared in the Wall Street Journal, Science, Scientific American, the Washington Post, BIOWorld, Congressional Quarterly and the Baltimore Sun, as well as on many TV and radio news programs. In 2013, he was named one of the Top 50 Global Stem Cell Influencers by Total BioPharma.

Want to read more Life Sciences Report interviews like this? Sign up for our free e-newsletter, and you'll learn when new articles have been published. To see recent interviews with industry analysts and commentators, visit our Streetwise Interviews page.

DISCLOSURE:
1) George S. Mack conducted this interview for Streetwise Reports LLC, publisher of The Gold Report, The Energy Report, The Life Sciences Report and The Mining Report, and he provides services to Streetwise Reports as an independent contractor. He owns, or his family owns, shares of the following companies mentioned in this interview: None
2) The following companies mentioned in the interview are sponsors of Streetwise Reports: None. The companies mentioned in this interview were not involved in any aspect of the interview preparation or post-interview editing so the expert could speak independently about the sector. Streetwise Reports does not accept stock in exchange for its services.
3) Morrie Ruffin: I own, or my family owns, shares of the following companies mentioned in this interview: None. I personally am, or my family is, paid by the following companies mentioned in this interview: None. My company has a financial relationship with the following companies mentioned in this interview: None. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
4) Michael Werner: I own, or my family owns, shares of the following companies mentioned in this interview: None. I personally am, or my family is, paid by the following companies mentioned in this interview: None. My company has a financial relationship with the following companies mentioned in this interview: None. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
5) Interviews are edited for clarity. Streetwise Reports does not make editorial comments or change experts' statements without their consent.
6) The interview does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer.
7) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their families are prohibited from making purchases and/or sales of those securities in the open market or otherwise during the up-to-four-week interval from the time of the interview until after it publishes.

Streetwise – The Life Sciences Report is Copyright © 2014 by Streetwise Reports LLC. All rights are reserved. Streetwise Reports LLC hereby grants an unrestricted license to use or disseminate this copyrighted material (i) only in whole (and always including this disclaimer), but (ii) never in part..
Streetwise Reports LLC does not guarantee the accuracy or thoroughness of the information reported.
Streetwise Reports LLC receives a fee from companies that are listed on the home page in the In This Issue section. Their sponsor pages may be considered advertising for the purposes of 18 U.S.C. 1734.
Participating companies provide the logos used in The Life Sciences Report. These logos are trademarks and are the property of the individual companies.
101 Second St., Suite 110
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Tel.: (707) 981-8204
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Cell Therapy 2015: With Maturity Comes Promise

noreply@blogger.com (Lee Buckler) — Tue, 23 Dec 2014 23:30:00 +0000

Under agreement with StreetWise Reports, I'm pleased to share their recent coverage of several companies in the cell therapy sector.

Original Source: Tracy Salcedo-Chourré of The Life Sciences Report (12/23/2014)
http://www.thelifesciencesreport.com/pub/na/cell-therapy-2015-with-maturity-comes-promise

A number of new and innovative medical paradigms are being explored by companies in the life sciences sector, with one of the front-runners being cell therapy—a disruptive technology that has, in the past few years, progressed out of preclinical studies and into clinical development. To learn more about the transformative promise of regenerative medicine, and what investors might expect from companies working in the sector in 2015, The Life Sciences Report asked analysts Jason Kolbert of Maxim Group and Dr. Christopher James of Brinson Patrick to discuss the advances that could generate robust returns on investment in coming years.

Cell Therapy in a Nutshell
Cell therapies have been gaining momentum in the life sciences for more than half a century. The first cell therapies, bone marrow transplants, were performed in the 1960s, and since then the idea of using healthy cells to replace or regenerate diseased cells has expanded into a number of indications, from cancer to cardiac disease to cosmetic therapy. Today, a number of companies are engaged in both clinical and preclinical trials, and there is some consensus that cell therapies will be "mainstream" in the near future.

The new year could prove a bellwether for regenerative medicine. The sheer volume of companies, universities and financial institutions conducting or backing research and development in the field attests to its potential: The Alliance for Regenerative Medicine(ARM), an advocacy group working with legislators, the investment community and regulatory agencies to promote development of cell therapy technologies, has more than 170 members working in the field.

On the horizon: Australian cell-therapy company Mesoblast Ltd. (MSB:ASE; MBLTY:OTCPK) is positioning itself for expedited approval of several of its allogeneic (derived from the same species) cell therapy products in Japan in 2015, following passage of legislation in that country that expedites approval. And though it hit a significant speed bump last month, Dendreon Corp.'s (DNDN:NASDAQ) Provenge (sipuleucel-T), an autologous (derived from and returned to the same patient) cell therapy for metastatic castrate-resistant prostate cancer, was the first FDA-approved cell therapy, and has helped pave a path forward for additional approvals in the U.S.

To get a handle on what investors can expect, and an idea of which companies to keep an eye on, The Life Sciences Report turned to Dr. Christopher James, managing director and senior equity research analyst with Brinson Patrick Securities Corp. and Jason Kolbert, managing director, senior biotechnology analyst and head of healthcare research with Maxim Group. Here's what they had to say about what 2015 might bring in both the sector and for certain companies working in the space.

Great Expectations
Asked which specific indications could be most responsive to cell therapies, and about companies working in those therapy areas, Kolbert provided several names.

The next "major event" in the sector will be results in a Phase 2 trial in ischemic stroke being conducted by Athersys Inc. (ATHX:NASDAQ), with results expected in Q1/2015, the analyst said. A number of other trials are also slated to start in 2015, including NeoStem Inc.'s (NBS:NASDAQ) Phase 3 trial in acute myocardial infarction and Cesca Therapeutics Inc.'s (KOOL:NASDAQ) pivotal trial in critical limb ischemia.

Kolbert is also looking forward to two trials involving Mesoblast's own stem cell product, as well as results from Prochymal (remestemcel-L, human mesenchymal stem cells for intravenous infusion, acquired from Osiris Therapeutics Inc. [OSIR:NASDAQ]). The Mesoblast product is expected to enter a pivotal trial in back pain, and the company is currently enrolling a global trial in congestive heart failure with partner Teva Pharmaceutical Industries Ltd. (TEVA:NASDAQ). The Osiris product is expected to be redeveloped in the U.S. for graft-versus-host disease (GvHD). Prochymal has been conditionally approved in Canada and New Zealand for GvHD in children. In addition, Mesoblast is expected to release data in a Phase 3 Prochymal trial in Crohn's disease.

James believes the area where cell therapy—cell regeneration—could have the biggest impact is also one of the riskiest—in the central nervous system (CNS), treating disorders or injury of the brain or spinal cord. James sees applications in multiple sclerosis (MS) and spinal cord injury, to regenerate neuronal function, and in Parkinson's disease, to address the death of dopaminergic cells. He also likes ocular applications, for treatment of such conditions as age-related macular degeneration (AMD).

James has two cell therapy companies under coverage. Opexa Therapeutics Inc. (OPXA:NASDAQ), with its Tcelna platform technology, is developing therapies in both CNS and the eye. A Phase 2 trial in secondary progressive MS is expected to read out in mid-2016. The company is also developing OPX-212, an autologous T-cell immunotherapy, to treat neuromyelitis optica, an autoimmune condition that leads to blindness.

The second company, StemCells Inc. (STEM:NASDAQ), is in a Phase 2 trial in the treatment of cervical spinal cord injury. On Dec. 18, the company announced that it had transplanted its HuCNS-SC (purified human neural stem cells) into its first patient; James expects final data from that trial in late 2015. Results from the company's Phase 1/2 trial in dry AMD, are expected in Q1/2015—perhaps as early as January.
StemCells' Phase 2 trial in cervical spinal cord injury points out what James sees as one of the regulatory hurdles cell therapies will have to overcome as they progress through the U.S. Food and Drug Administration's (FDA's) approval process. The trial is a randomized, controlled, single-blind study, as opposed to the FDA standard of double-blind pivotal studies.

To treat central nervous system disease or injury, companies must inject cells into a patient, a "surgical" intervention, James explained. But clinicians "can't do a sham surgery on the brain or on the spine. . .that's not ethical." So companies like StemCells must work with the FDA to develop pivotal trial designs that are "less stringent" than the double-blind model.

The Bigger Picture
Kolbert is enthusiastic about the prospects for cell therapies in 2015, and doesn't see much in the way of headwinds. "Cell therapy ultimately lowers the cost of treating disease, and at a time when there is an outcry over high-priced therapies, cell therapy offers promise to lower the cost of treating disease. In terms of politics, the cell therapies we are focusing on are typically not controversial (using adult stem cells, not cells from embryos). As such, we don’t see any controversy. The big change in the landscape now is the acceptance that cell therapy is 'safe.'"

On the plus side of the spectrum is oncology. Using cell therapy in cancer immunotherapy "holds great promise," Kolbert continued. "The ability to rev up the immune system with checkpoint inhibitors, and couple that with therapeutics, like the therapies being developed by ImmunoCellular Therapeutics Ltd. (IMUC:OTCBB) and Agenus Inc. (AGEN:NASDAQ) targeting glioblastoma, and the work that OncoSec Medical Inc. (ONCS:OTCBB), Inovio Pharmaceuticals Inc. (INO:NYSE.MKT) and NeoStem are doing in melanoma, is exciting."

James and Kolbert agree that recent elections, in which Republicans gained control of both houses of Congress, shouldn't affect ongoing work in the cell therapy field. "They have bigger issues to deal with," James commented.

However, James believes cell therapy companies may suffer from an "overhang" following the recent bankruptcy of Dendreon Corp. And Kolbert cautions investors that second- and third-generation technologies always follow. Such is the case with Bavarian Nordic's (BAVA:OMX) Prostvac. Prostvac has the potential to be equivalent to, or even superior to, Provenge, but at a fraction of the cost. The Phase 3 Prostvac trial reports data next year.

Another concern for investors, according to James, is the question of whether cell therapy companies can manufacture their products on a commercial scale. But the analyst believes that concern can be addressed, and cites the StemCells model, in which, according to the company, "[c]ryopreserved lines of donor-derived cells can be reproduced at commercial scale as 'stem cells in a bottle,' then distributed for patient doses as needed, much like an off-the-shelf pharmaceutical product."

Christopher James, M.D., is a managing director and senior equity research analyst focusing on life sciences companies with strong growth potential and developing novel agents for serious diseases including cancer and infectious, neurological, inflammatory, metabolic and cardiovascular diseases. He was previously a senior equity research analyst at Rodman & Renshaw and MLV & Co. Prior to joining Brinson Patrick, Dr. James was chief medical officer and senior vice president of medical affairs at Retrophin, a biotechnology company focused on developing therapeutics for rare and devastating diseases. While at Retrophin, he played a pivotal role in the study design and subsequent acceptance of an investigational new drug application with the cardiorenal division of the FDA to initiate a Phase 2 clinical study in a rare kidney disease called focal segmental glomerulosclerosis. Dr. James has prior buyside experience working at Trivium Capital Management and MSMB Capital Management. Dr. James trained in neurological surgery at Cornell-New York Hospital and Memorial Sloan Kettering Cancer Center. He obtained a medical degree from Yale University School of Medicine and a bachelor of science in biology from Cornell University.

Jason Kolbert has worked extensively in the healthcare sector as product manager for a leading pharmaceutical company, as a fund manager and as an equity analyst. Prior to joining Maxim Group, where he is head of healthcare research, senior managing director and biotechnology analyst, Kolbert spent seven years at Susquehanna International Group, where he managed a healthcare fund and founded SIG's biotechnology team. Previously, Kolbert served as the healthcare strategist for Salomon Smith Barney. He is often quoted in the media and is a sought-out expert in the biotechnology field. Prior to beginning his Wall Street career, Kolbert served as a product manager for Schering-Plough in Osaka, Japan. He received a bachelor's degree in chemistry from State University of New York, New Paltz, and a master's degree in business administration from the University of New Haven.

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DISCLOSURE:
1) Tracy Salcedo-Chourre compiled this article for Streetwise Reports LLC, publisher of The Gold Report, The Energy Report, The Life Sciences Report and The Mining Report, and provides services to Streetwise Reports as an employee. She owns, or her family owns, shares of the following companies mentioned in this interview: None.
2) The following companies mentioned in the interview are sponsors of Streetwise Reports: NeoStem Inc., Athersys Inc., StemCells Inc., Inovio Pharmaceuticals Inc. Mesoblast Ltd. is not affiliated with Streetwise Reports. The companies mentioned in this interview were not involved in any aspect of the interview preparation or post-interview editing so the expert could speak independently about the sector. Streetwise Reports does not accept stock in exchange for its services.
3) Christopher James: I own, or my family owns, shares of the following companies mentioned in this interview: None. I personally am, or my family is, paid by the following companies mentioned in this interview: None. My company has a financial relationship with the following companies mentioned in this interview: Opexa Therapeutics. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
4) Jason Kolbert: I own, or my family owns, shares of the following companies mentioned in this interview: None. I personally am, or my family is, paid by the following companies mentioned in this interview: None. My company has a financial relationship with the following companies mentioned in this interview: None. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
4) Interviews are edited for clarity. Streetwise Reports does not make editorial comments or change experts' statements without their consent.
5) The interview does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer.
6) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their families are prohibited from making purchases and/or sales of those securities in the open market or otherwise during the up-to-four-week interval from the time of the interview until after it publishes.

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Reprint from LSR: How RepliCel Is Harnessing the Awesome Power of Cell Therapy

noreply@blogger.com (Lee Buckler) — Fri, 07 Nov 2014 19:17:00 +0000

I've interviewed a number of executives from several cell therapy companies recently and intend to post my interview of them in the coming weeks but in the interim, StreetWise Reports published this interview of me which I'm pleased to share.

Original Source: George S. Mack of The Life Sciences Report (11/07/2014)

Regenerative medicine and cell therapies hold possibilities for achieving near miracles in a multitude of indications, from life-saving treatments to aesthetic applications. RepliCel Life Sciences Inc. (RP:TSX.V; REPCF:OTCQB) is tackling a mix of medical and cosmetic issues that include hair regeneration, repair of painful and debilitating tendon injuries and rejuvenation of damaged skin. In this interview with The Life Sciences Report, R. Lee Buckler, the company's new vice president of business and corporate development, discusses his firm's innovative technology platform and the upcoming milestones that could affect its shares.

The Life Sciences Report: Lee, you've made a recent career change and are now an executive at RepliCel. Tell us about that.

R. Lee Buckler: As of Oct. 1, I have been appointed vice president of business and corporate development for RepliCel Life Sciences Inc., which is engaged in development of cell-based regenerative medicine therapeutics in Canada, Europe and Japan through our licensing partner, Shiseido Company Ltd. (4911:TSE). I found RepliCel to be a very interesting company poised to go on an exciting run, and that enticed me to join the team.

TLSR: Prior to your work with RepliCel, what experience had you had in the cell therapy industry?

RLB: In 2000, I left the practice of law to join Allen Eaves in the Stem Cell Technologies group of companies, where I ran a company called Malachite Management Inc. In 2006, I was recruited by Progenitor Cell Therapy to run its business development, marketing, communication and sales before the NeoStem acquisition.

In 2008, I founded my own consulting firm, called the Cell Therapy Group (CTG), which focused exclusively on the cell therapy industry. In the early days, we did some communications work for clients, but for the bulk of my tenure with CTG, we were involved in a wide range of planning and business development work. Some of it was transactional, but other aspects included market and competitive intelligence, building strategies, identifying partners, targeting partners, engaging in partnership discussions on behalf of clients and the like. I also worked with fund managers and investors through education, namely technology and platform explanations.

Over the past year, I worked as a consultant and on the board of directors positioning TheraVitae Inc. (private) for a merger with a company listed on the Toronto Venture Exchange. The merger is expected to complete in early November, and the company will be renamed Hemostemix Inc. I helped TheraVitae raise several million dollars as part of that process. Being on the road giving presentations to prospective investors is a new skill set for me, but I've found I really enjoy this side of the business.

TLSR: You are an attorney by training, but I see from your curriculum vitae that you did a couple of stints as a medical laboratory technician while you were still in law school. Is that what led you to the life sciences field?

RLB: Yes. I always joke with people that I didn't get to the cell therapy/regenerative medicine industry through education—I got here more by osmosis. I was not a silver spoon kid; I had to work my way through school. So while I was studying to be an attorney, I ended up working in the lab of a leading cardiovascular investigator, who was involved in some clinical trials at the time. I was mainly doing grunt work, but it exposed me to an environment where people were extremely dedicated to their sciences and to doing something novel. I was exposed to the excitement that builds when people truly believe what they're doing could revolutionize the way people are treated.

I've always felt a little bit like an outsider in an industry of people who belong here. While that may feel uncomfortable from time to time, it also affords me a unique perspective that others don't have. While others in the industry tend to focus on vertical specialties, I've come to specialize in a macro view of this industry. My focus has been very horizontal, which gives me a perspective of the industry that not many people are able to see.

TLSR: What kind of work has RepliCel been doing in the cell therapy field?

RLB: When CEO and President David Hall took over the company in 2011, it was built around hair regeneration. That is still an important part of our portfolio; however, he had a vision for broadening the technology and building a platform, which the company has now executed.

We are preparing to launch a very significant Phase 2 trial using our RCH-01 (dermal sheath cup [DSC] cells) for hair regeneration. This is a cellular injection—a cell transplant rather than a hair transplant—and is an important evolution because hair transplant is limited by three very significant factors. First, when transplanting hair follicles from one location on your scalp to another, there are only so many follicles available to harvest. With a cell transplant, there is no limit to the number of cells we can grow to use in regenerating poorly functioning hair follicles. Second, hair transplantation only achieves a satisfactory result when performed by a gifted surgeon, of which there are few. A simple cell injection takes the art out of the procedure—particularly when combined with our proprietary injection device designed to optimally deliver the cells into the scalp. Finally, hair transplantation is not an option women find attractive for a number of reasons, and a significant population of women suffer from hair loss.

TLSR: How is RepliCel working to ensure this therapy will be effective in both the short and long term? What prevents the dermal sheath cells from ceasing to grow hair once they are in the locale where the original follicles quit producing hair?

RLB: The cells we are using to address pattern baldness (androgenetic alopecia) are taken from a cell population found at the base of the hair follicle. These DSC cells are used to produce our RCH-01 product. Research has demonstrated that these cells are responsible for the reorganization of the hair follicle, which is a mini-organ that organizes upon an unknown signal. Our research leads us to believe this cell population is responsible for hair regeneration.

We source our particular cells from hair follicles isolated from the back of the scalp, between the ears, because most balding people retain this area of hair. This hair is insensitive to the androgen hormone (DHT), which causes hair loss, making these hair follicles prime candidates for our hair regeneration product.

As to the question of whether this will be a durable response—how long the hair will last—this is one of several questions both we and Shiseido are targeting in our respective upcoming pattern baldness trials. We're designing this next phase to look at dosing. We're also looking at frequency of treatment: One cohort of the study gets a single treatment, another gets a second treatment at day 91.

But we'll also be following these patients for a considerable length of time, to see whether the intended effects are maintained or whether they diminish over time. Even though there are only a proposed 160 participants, utilizing different dosing and different injection points throughout the scalp, there will be 396 treatment sites, or data points, that we will be able to gather from those 160 patients, in addition to the data to be gleaned and shared from the trial Shiseido is funding in Japan. These questions are great—effectiveness and duration of effect—and we have an obligation to answer them, which is why the trials are designed the way they are.

TLSR: What else is RepliCel working on at the moment?

RLB: We have another population of cells derived from the hair follicle (the non-bulbar dermal sheath cells [NBDS cells]) that we believe is a platform capable of generating multiple products for various indications. These cells can be readily expanded, and it turns out they are highly expressive of type 1 collagen. Our first trial with these cells will be using our RCT-01 product for the treatment of chronic Achilles tendinosis.

Up to 90% of healthy tendon is comprised of well-constructed type 1 collagen, and a number of indications manifest in patients due to the loss of type 1 collagen production in the endogenous cells, one of which is Achilles tendinosis. Tendons often are not well vascularized, and after a series of injuries and as a patient ages, the endogenous fibroblasts are exhausted of their ability to continue to produce the type 1 collagen necessary to support healthy tendons. RepliCel's predicate science is built around the injection of autologous (harvested and administered back to the same patient) fibroblasts capable of producing the kind of collagen needed to restore the patient to healthier function and better pain scores.

TLSR: Do you have evidence of actual tendon regeneration?

RLB: In three tendinosis-related clinical trials performed using a similar cell type, which has now been licensed into the company, MRI imaging shows that tendon treated with this cell type was much more akin to healthy, young, functioning tendon than what the patients had prior to injection of cells. This is an exciting platform, and the company is about to launch a Phase 1/2 trial in chronic Achilles tendinosis. We believe the cells could also have application in other indications, including jumper's knee, golfer's elbow, tennis elbow and torn rotator cuffs, as well as in a number of dermatological applications. Late this year, we will launch a Phase 1 study in healthy volunteers to look at the ability to regenerate the underlying tissue of skin in patients who have aging or sun-damaged skin.

TLSR: Achilles tendinosis and androgenic alopecia are very different indications. Androgenic

alopecia is a hormone-dependent condition, while Achilles tendinosis is trauma-related.

RLB: That's a great point. Even though both of these studies—tendon repair and hair regeneration—use cells derived from the hair follicle, we're working with two very different cell populations. As a result, they have the ability to elicit very different, targeted responses.

TLSR: RepliCel's shares have suffered considerably over the past six months. What caused the dip and what is the company doing to fix the issue?

RLB: The fact of the matter is the company was delayed in progressing to its Phase 2 trial for RCH-01 in hair regeneration because of an issue with the supply of a critical growth media. The new media wasn't producing the same results, so we had to go back to the drawing board and discover what the problem was. The comparability data is now coming in to support our belief that we've solved that problem. We have four trials expected to launch in the next few months (three of ours and one of Shiseido's). Two of these are expected to give clinical readouts late next year. Until we are a company executing clinical trials, we are a company talking about executing clinical trials, and certain investors grow understandably impatient.

I'm very pleased that in October we submitted an application to Health Canada for the proposed Phase 1/2 clinical trial for chronic Achilles tendinosis. This triggers a 30-day window during which Health Canada can provide a No Objection Letter allowing us to proceed with the trial. This event is the initial trigger for a cascade of catalysts anticipated over the following months related to this trial, as well as our proposed Phase 1 clinical trial in Germany for aging and sun-damaged skin, our proposed Phase 2 trial for pattern baldness (androgenetic alopecia) in Germany, and Shiseido's upcoming trial for pattern baldness in Japan. We've been on the road for several weeks, crisscrossing the U.S., Canada and Japan, talking to investors, analysts and potential partners. We are pleased with the level of interest being generated, and believe that once we demonstrate we are executing on schedule, we will generate increased support.

TLSR: Is it possible that all these milestones will be met by the end of 2014?

RLB: We are working very hard to make that happen. I believe we're on target to have our three clinical trial application filings submitted by year-end. We expect Shiseido to file its clinical trial application early in Q1/15.

We are, of course, dependent on regulatory clearance to initiate any trial, but we have had very active dialogues with the regulators overseeing all our proposed trials, and are submitting precisely what has been discussed. As mentioned, we have now filed the first of our three applications. The second trial application proposes to use a product (RCS-01) developed from the same platform technology, so the clinical-regulatory team can leverage much of the work already done to get the second application filed.

One thing to note is that the dermatology and tendinosis trials are relatively quick studies to enroll. We've been in constant dialogue with the principal investigators of the RCT-01 trial in chronic Achilles tendinosis, and they assure us there's a pipeline of patients waiting to enroll in the study. The RCT-01 tendon trial is going to be a 28-participant study, and the RCS-01 in skin rejuvenation is a proposed 28-participant study design as well, but using healthy volunteers. The RCH-01 hair regeneration study is going to extend over a longer period of time, because it targets 160 participants. But the RCT-01 and RCS-01 studies will be relatively quick to enroll and to follow up on, and we expect data in 2015 for both of those.

Both of these studies are randomized, placebo-controlled and specifically designed to provide measurable and material biologic and mechanistic data that we will use to drive partner discussions. Remember that the company's business model is to codevelop assets with partners who understand the markets and have proven commercialization capabilities.

We are excited about being in the position we are now in, poised to imminently execute on three clinical trials, finalize the development and validation of our propriety injection device (which has licensable applications for acellular injectables), capitalize on our partnership with Shiseido and the innovative regulatory pathway for regenerative medicines in Japan, which provides a window to early-market access for our pattern baldness treatment, and to execute on one or more additional licenses with co-development partners in the near term.

TLSR: Thank you very much for your insight, Lee.

RLB: Thank you.

R. Lee Buckler is vice president of business and corporate development with RepliCel Life Sciences Inc. Prior to working with RepliCel, he was the managing director of Cell Therapy Group, a firm he formed in 2008, where he did business development consulting for companies and organizations in or interested in the cell therapy sector. Buckler served six years as executive director of the International Society for Cellular Therapy, and just over two years as director of business development for Progenitor Cell Therapy. He is on the editorial advisory boards of the journal Regenerative Medicine and the BioProcess International magazine, as well as the co-chair of the Alliance for Regenerative Medicine's Communications and Education Committee. Buckler cofounded Cell Therapy News, founded Cell Therapy Blog, cofounded Regenerative Medicine Jobs, founded and continues to manage the LinkedIn Cell Therapy Industry Group, and is an active industry commentator in publications and in social media. He serves on numerous industry conference advisory boards, is an advisory board member for BioCision and RoosterBio, and is on the board of directors for Hemostemix. He has a bachelor's degree in education and a law degree.

For additional comments on RepliCel Life Sciences Inc., Shiseido Company Ltd. and Hemostemix Inc. from newsletter writers, money managers and analysts, click on their respective links or visit The Life Sciences Report.

Want to read more Life Sciences Report interviews like this? Sign up for our free e-newsletter, and you'll learn when new articles have been published. To see recent interviews with industry analysts and commentators, visit our Streetwise Interviews page.

DISCLOSURE:

1) George S. Mack conducted this interview for Streetwise Reports LLC, publisher of The Gold Report, The Energy Report, The Life Sciences Report and The Mining Report, and provides services to Streetwise Reports as an independent contractor. He owns, or his family owns, shares of the company mentioned in this interview: None.

2) RepliCel Life Sciences Inc. paid Streetwise Reports to conduct, produce and distribute the interview

3) R. Lee Buckler had final approval of the content and is wholly responsible for the validity of the statements. Opinions expressed are the opinions of R. Lee Buckler and not of Streetwise Reports or its officers.

4) The interview does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their families are prohibited from making purchases and/or sales of those securities in the open market or otherwise during the up-to-four-week interval from the time of the interview until after it publishes.

Streetwise Reports LLC does not guarantee the accuracy or thoroughness of the information reported.

Participating companies provide the logos used in The Life Sciences Report. These logos are trademarks and are the property of the individual companies.

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Japan Gives a Hug to the Cell Therapy Industry

noreply@blogger.com (Lee Buckler) — Fri, 24 Oct 2014 16:21:00 +0000

Original Source: George S. Mack of The Life Sciences Report (10/22/14)

Last year, Japan rolled out the red carpet for cell therapy developers with new legislation designed to expedite development of regenerative medicine solutions for all manner of disease. In this interview with The Life Sciences Report, former regenerative medicine consultant R. Lee Buckler, now a vice president with RepliCel Life Sciences Inc., puts his consultant's hat back on to talk about Japan's hospitable stance on proposed cell therapies. He provides an update on the meaning of the new law and discusses a small cohort of companies poised to reap the advantages.

The Life Sciences Report: (TLSR) Lee, you've been around the stem cell and regenerative medicine industry for quite a long time, and you have a special interest in the new legislation enacted by the Japanese Diet (parliament) back in November 2013. The intention of the law is to hasten clinical development of cell therapies and regenerative medicine therapies. A new cell therapy can get conditional approval in Japan for a limited time with clinical (Phase 2) data showing safety in humans. This new law goes into effect in November. Tell me your thoughts.

R. Lee Buckler: The entire industry is watching Japan and this new regulatory model with an incredible amount of interest. We're on the brink of seeing more detail around the regulations.

This initiative sets up a paradigm of conditional approval, where a company can bring an application to the Japanese regulators, and if it shows sufficient safety evidence and some evidence of efficacy, the company could get conditional approval to sell its product in the Japanese marketplace. This conditional approval would be for a seven-year window, during which the sponsor or sponsors would have an obligation to continue to file clinical data toward an eventual final market approval—a biologics license application (BLA) or the Japanese equivalent thereof. There is also some suggestion that during the seven-year window, some reimbursement will be attached to the product being sold on the market.

TLSR: In mid-September, Pluristem Therapeutics Inc. (PSTI:NASDAQ), an Israeli company, said it was going to pursue a development strategy in Japan. It hired consultants to help the company get into that market. The company's lead indications are peripheral artery disease, intermittent claudication, critical limb ischemia and muscle injury. But otherwise, I don't see a lot of other companies initiating programs in Japan. Do you see companies from the U.S., Canada or Europe getting into that regulatory environment?

RLB: I think most companies in the regenerative medicine industry have a tremendous desire to have a Japanese strategy right now. The problem is that a lot of companies in this sector don't have enough bandwidth, or capital, to execute on that.

However, a few are making Japan a high priority. Currently, less than a handful of companies have a clearly articulated Japanese strategy, and only that many again have begun executing on the opportunity.

Pluristem has made the announcement about pursuing opportunities in Japan, and Chairman and CEO Zami Aberman has done an admirable job of executing deals. The company has the partnership with United Therapeutics Corp. (UTHR:NASDAQ), which is conducting a Phase 1 study of the company's PLX-PAD (full-term placenta-derived adherent stromal cells) product in pulmonary arterial hypertension, which is a fatal disease and happens to be a very large market. Pluristem also has a nice deal in place in Korea with CHA Biotech Co. Ltd. (CHA:KOSDAQ).

Going it alone all the way to market is not a good option for most cell therapy companies, and with the change in the Japanese regulation there is an appetite for regenerative medicine products in Japan that foreign companies in the sector need to capitalize on.

TLSR: You said there were a few others. Give me an overview, please.

RLB: Athersys Inc. (ATHX:NASDAQ) has announced a desire to move into Japan, and I know it is putting boots on the ground in the country to develop a strategy there. Back in January, the company announced some new Japanese patents in graft-versus-host disease (GvHD) and autoimmune diseases, such as inflammatory bowel diseases.

Cytori Therapeutics Inc. (CYTX:NASDAQ) has always had strong relationships in Japan, and has been there for a decade. It has a subsidiary in Tokyo called Cytori Therapeutics K.K.

Also, my own company, RepliCel Life Sciences Inc. (RP:TSX.V; REPCF:OTCQB), has an existing relationship with Shiseido Company Ltd. (4911:TSE), which will launch a clinical trial in Japan for pattern baldness in the next few months. This is one of the few cell therapy deals—perhaps the only one to date—that involves putting manufacturing capacity in Japan.

The company that is potentially in the pole position in Japan at the moment, however, may be Mesoblast Ltd. (MSB:ASE; MBLTY:OTCPK). The company inherited a relationship with Japan-based JCR Pharmaceuticals Co. Ltd. (4552:TKY) when, in 2013, it acquired the Prochymal (remestemcel-L or allogeneic, adult human mesenchymal stem cells) product portfolio from Osiris Therapeutics Inc. (OSIR:NASDAQ). The Osiris/JCR partnership, which was put into place a number of years ago, had been stagnant until the reemergence of the Japanese market as an important one for the regenerative medicine sector. Now Mesoblast has "regenerated" that relationship, and announced on Oct. 1 that JCR would be filing a market approval application for Prochymal in Japan for the treatment of pediatric GvHD, following similar approvals in Canada and New Zealand.

TLSR: Will a company be able to take Phase 2 data generated in the U.S. or Europe or Canada, including positive proof-of-concept and safety data, and submit that in Japan, potentially getting a seven-year conditional approval?

RLB: That's an important question. The best intelligence I have, based on interacting with people who have been working with the Japanese, including Professor Chris Mason from University College London, indicates the answer is "yes." The Japanese government is intentionally welcoming ex-Japan data for conditional market approval in Japan.

If a company gets conditional approval to market a product in Japan, it will also have to generate pivotal data outside of Japan, because no patients in Japan will want to risk being involved in a placebo trial when they have the option to buy the product in the marketplace. The end result of the paradigm is that there will continue to be data generated outside of Japan, which will be brought into Japan to support eventual final approval.

TLSR: Thank you for your time.

RLB: Many thanks to you.

[Editor's Note: Between the time this interview was scheduled and the time it was conducted, Buckler left his consultancy firm to become vice president of business and corporate development at Vancouver-based RepliCel Life Sciences Inc., a cell therapy company.]

R. Lee Buckler, vice president of business and corporate development at RepliCel Life Sciences Inc., has been an executive in the cell therapy sector since 2000, beginning with Malachite Management in the Stem Cell Technologies group of companies. Most recently he was the managing director of Cell Therapy Group, a firm he formed in 2008 to do business development consulting for companies and organizations working in or interested in the cell therapy sector. His work included deal-targeting, transactions, market intelligence, competitive analyses, strategic assessments and market profile planning for companies ranging from top-tier multinationals to start-ups. Buckler served six years as executive director of the International Society for Cellular Therapy and just over two years as director of business development for Progenitor Cell Therapy. Buckler has a bachelor's degree in education, and a law degree. He is on the editorial advisory boards of the journalRegenerative Medicine and the BioProcess International magazine. He is also co-chair of the Alliance for Regenerative Medicine's Communications and Education Committee. He is an active industry commentator in publications and in social media and serves on numerous industry advisory boards.

DISCLOSURE:

1) George S. Mack conducted this interview for Streetwise Reports LLC, publisher of The Gold Report, The Energy Report, The Life Sciences Report and The Mining Report, and he provides services to Streetwise Reports as an independent contractor. He owns, or his family owns, shares of the following companies mentioned in this interview: None.

2) R. Lee Buckler: I own, or my family owns, shares of the following companies mentioned in this interview: RepliCel Life Sciences Inc. I personally am, or my family is, paid by the following companies mentioned in this interview: RepliCel Life Sciences Inc. My company has a financial relationship with the following companies mentioned in this interview: None. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I determined and had final say over which companies would be included in the interview based on my research, understanding of the sector and interview theme. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.

3) The following companies mentioned in the interview are sponsors of Streetwise Reports: RepliCel Life Sciences Inc., Athersys Inc. Mesoblast Ltd. is not affiliated with Streetwise Reports. The companies mentioned in this interview were not involved in any aspect of the interview preparation or post-interview editing so the expert could speak independently about the sector. Streetwise Reports does not accept stock in exchange for its services.

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Lessons from Toronto on the Future of Regenerative Medicine in Japan

noreply@blogger.com (Lee Buckler) — Thu, 24 Jul 2014 21:27:00 +0000

One of the early pioneers of cell therapy as we know it today is Professor Arnold Caplan commonly thought of as responsible for the identification and isolation of the mesenchymal stem cell. Several years ago Dr. Caplan launched a summer course at Case Western Reserve University he called Business of Regenerative Medicine. This year it was hosted by the Centre for Commercialization of Regenerative Medicine (CCRM) in Toronto - the first time it's been hosted outside of Cleveland.

I was fortunate to be invited to participate in this year's 7th Annual “Business of Regenerative Medicine: New Therapies, New Models”. Signals blogger, Mark Curtis, has done a nice job of summarizing the highlights of this year's course.

One of the most valuable take-aways for me was from a talk given by the inimitable Professor Chris Mason of University College London who has reportedly been advising Japanese lawmakers and regulators on their new proposed regulatory framework for regenerative medicine.

Let me back up. In June 2012, regenerative medicine was given priority status in the national Cabinet Secretariat’s Five-year Healthcare Innovation Strategy. In October 2012 Prof. Shinya Yamanaka of Kyoto University won a share in the Nobel Prize in Physiology or Medicine for his work in the development of iPS cell technology. This solidified regenerative medicine as an area of national pride and one of the areas in which Japan wanted to demonstrate innovative global leadership not just for scientific discovery but for the development of an industry around those discoveries. However, the Japanese regulatory framework which applied to cell therapies was widely recognized as being in desperate need of revision in order to support true commercialization of these products. It made sense, then, that the Japanese started their innovation with the regulation.

In November 2013, the Japanese legislature (the National Diet) passed legislation that revised Japan's Pharmaceutical Affairs Law and created the new Regenerative Medicine Law intended to be the groundwork for establishing Japan as a global leader in regenerative medicine in the years ahead. The import of this legislation was that it created a legislative mandate to create regulation that established an expedited development and commercialization pathway for regenerative medicine therapies while continuing to ensure and protect patient safety. The legislation passed with overwhelming support in both the lower and upper houses of the Diet. Since then the policy makers and regulators at the MHLW and PMDA have been busily attempting to build a regulatory framework around this mandate.

There has been a lot written about this potentially ground-breaking approach to regulating cell therapies (see also here and here) but until now I have seen very little detail around how the mandate of the new law might be translated into operational regulation. On that front, here is my brief second-hand synopsis of what I think I learned from Chris:

conditional approval will be available to any cell therapies that provide evidence of safety and early signals of efficacy - this is reportedly not limited in any way by the indication or by product type

in other words there is no need to be addressing an orphan indication or for the product to be autologous in order to qualify
at least in the early days of this implementation it is expected that this data will be 'imported' from trials conducted outside of Japan

once conditional approval is granted, a sponsor company will have 7 years to obtain final approval

there is likely to be a requirement that a product be manufactured in Japan to obtain final approval

conditional approval will also come with reimbursement though there is no indication yet as to how pricing will be determined
there is an expectation that the pivotal studies needed to obtain final approval will be done outside of Japan - since the product is already being sold in Japan, conducting a placebo-controlled trial would be next-to-impossible

There are a number of Japanese companies already in the regenerative medicine and cell therapy space that may be the initial and primary beneficiaries of this new regulatory paradigm. Most of these are involved in the Forum for Innovative Regenerative Medicine (FIRM) established in 2011 to promote and support the commercialization of regenerative medicine in Japan.

As largely an industry organization, FIRM really started emerging as a key national player with its involvement in the development of this newly emerging regulatory framework. To quote FIRM, "Commercializing this state-of-the-art technology is not just a question of scientific and biotechnological prowess; the broad-ranging manufacturing capabilities nurtured by Japan’s industrial community will also be an indispensable element. Indeed, this is an opportunity for Japan to leverage its unique technologies to grow an internationally competitive industry."

Over the past months it has been interesting to observe how companies in Japan (even large multinational conglomerates) have responded to this new national mandate. Many of them appear quite anxious to demonstrate to their shareholders, government, and peers that they have active programs in regenerative medicine and thus are supporting this national initiative. This appears to be creating considerable appetite among Japanese companies, many of whom have not been in the sector to-date, for regenerative medicine technologies with which they can associate.

As a result of this phenomenon, we expect there will be many regenerative medicine companies outside of Japan which will find wins for themselves as a result of this national initiative. Naturally, companies in the regenerative medicine and cell therapy space outside of Japan are tripping over themselves to prioritize Japan as part of their corporate planning and to elucidate their Japanese strategy to investors and partners as a competitive advantage and potential earlier route to market if not return on investment. Some of the companies distinguishing themselves early in this race are Cytori (NASDAQ:CYTX), Mesoblast (ASX:MSB), and Athersys (NASDAQ:ATHX).

Cytori has long been in Japan and is making sure no one forgets that by press releasing the importance of their Japanese focus.

Mesoblast inherited a Japanese licensee when they took over the Prochymal portfolio from Osiris. JCR Pharmaceuticals has the license for the allogeneic mesenchymal stem cell (MSC) product in Japan to treat steroid refractory graft-versus-host disease (GvHD). Mesoblast has announced its intention to leverage this door to the Japanese market. Given that the product has already received approval in Canada and New Zealand for pediatric GvHD, it has been speculated that JCR may be the first company to secure conditional approval for a cell therapy in Japan.

Not to be outdone, Athersys announced in January, "We are actively evaluating the potential for accelerated development of MultiStem cells in a number of therapeutic areas in Japan, and will seek to establish strategic collaborations to assist in the rapid development of products that will help Japanese patients."

One of the sleepers in this race may be an emerging regenerative medicine company named RepliCel Life Sciences (TSXV:RP). In May 2013 RepliCel announced a Collaboration and Technology Development Transfer Framework Agreement with Shiseido Company, Limited (Tokyo Stock Exchange Code: 4911) for an exclusive geographic license for RepliCel’s RCH-01 hair regeneration technology. Shiseido has an exclusive geographic license to use RCH-01 in Japan, China, South Korea, Taiwan and the ASEAN countries.

This deal garnered RepliCel ~$4M in upfront cash and was valued at over $30M in milestone payments and sales royalties. Perhaps more importantly, since that time the companies have executed a tech transfer agreement, RepliCel was granted a key patent in Japan, and Shiseido recently celebrated the opening of its new cell-processing facility at the Kobe Biomedical Innovation Cluster (KBIC). This facility will focus on the commercialization of RepliCel’s RCH-01 hair regeneration technology for Shiseido's markets.

In speaking with management, I understand Shiseido has plans to conduct its own clinical trial of the product in Japan and that the companies are collaborating closely (through a joint development committee) on the continued improvement of the technology to ensure a single product emerges. This means there will be data from two concurrent trials (RepliCel's proposed phase 2 trial in Germany and Shiseido's proposed trial in Japan).

My own take on this is that it seems RepliCel/Shiseido may be well positioned to take advantage of early conditional approval of this technology in Japan in a way that generates near-term revenue while they continue to generate clinical data and optimize the treatment (dose, frequency) and manufacturing. In due course this data will be available to RepliCel to use in licensing discussions for non-Shiseido markets. Furthermore, RepliCel/Shiseido have already solved the anticipated hurdle of having manufacturing in the country as a predicate to eventual final approval.

The fact that their Shiseido collaboration may position RepliCel as a leader among foreign companies taking early advantage of this new regenerative medicine framework in Japan has not been lost on management (see here) but given RepliCel's expanded technology platform and product/clinical pipeline combined with the 3rd party validation and unique attributes of the Shiseido deal, I'm not sure the message has sunk in with investors yet given the ~$30M market cap.

I'll be watching the Japanese regenerative medicine race with great interest over the next few months. It has been reported that there will be a MHLW-sponsored public comment period for the new regenerative medicine law announced soon. All stakeholders are encouraged to provide feedback on the new legislation aimed at regenerative medicine therapies.

Also of note will be to monitor how other regulatory agencies respond. Already we have seen moves toward expedited or conditional approvals (in limited circumstances) in Europe and potentially expedited pathways (e.g., breakthrough designation) in the U.S. Other jurisdictions such as South Korea, Canada, and Australia have apparently taken a more functional approach to expediting certain cell therapy product approvals but on a case-by-case basis.

RepliCel -- Treating Functional Cellular Deficits. An Interview with CEO, David Hall

noreply@blogger.com (Lee Buckler) — Tue, 27 May 2014 06:25:00 +0000

Despite us both living in the Vancouver area and both being involved in cell therapy, I didn't meet David Hall until we were both in La Jolla late last year for the Alliance for Regenerative Medicine's Stem Cell on the Mesa meeting. Since then we've been keeping in close contact and I have had opportunity to learn a lot more about him, his team, RepliCel, and the company's technology.

The company is by all appearances a company on the tail end of a number of significant transitions in terms of its structure, finance, clinical pipeline, and even the breadth of its underlying technology. The company is now unveiling RepliCel 2.0 (my term, not theirs) which builds on the company's core expertise and platform technology but combines newly added assets and clinical strategies that create end-to-end solutions and multiple pillars around which shareholder value is being created.

David presents a sound logic for the transitions through which he has taken the company in the past 18 months and the pace of that change. He does, perhaps more than many CEO's I have had the opportunity to interview, present a strong and focused vision for the company with a clear goal and a well-defined pathway for how he intends to get the company there. From my vantage point, this vision permeates into most aspects of the company from clinical trial design, to patent strategy, to defining collaborations, and even recruiting.

Suffice it to say I have been increasingly impressed by what I see and welcome the opportunity to share the interview below with you. I hope you enjoy learning a little more about RepliCel as much as I have.

CTB: Tell us a little about your background, David, and how you got involved in a cell therapy company?

DH: My initial career was in finance working on both sides of the street as a money manager and as a banker and analyst. It was as a banker that I came to join ID Biomedical which then led to my joining Angiotech Pharmaceuticals not long after it was started. During my time there I served as Chief Financial Officer, Chief Compliance Officer and Treasurer and Corporate Secretary. After 20 years in life sciences, I spent time focused on public policy in BC and Canada as it related to developing our BC life sciences industry. During that time I was approached to look at a cell therapy company developing a treatment for pattern baldness. Despite my lack of empathy at that time for pattern baldness as a disease, I was struck by the elegance of this group’s scientific approach and how that might be expanded into other treatments where there was a deficit of healthy active cells.

CTB: Can you give us a snapshot of RepliCel as a company?

DH: RepliCel is treating cellular deficits in two main areas. The first is addressing conditions where there is a deficit of healthy functioning fibroblasts such as chronic tendinosis and damaged skin.

The second is in treating pattern baldness where there is a deficit of dermal sheath cup cells that are responsible for maintaining a hair follicle cycle of fiber production.

In addition, we have developed cell manufacturing technology and procedures to support the potential commercialization of these indications as well as specialised delivery devices which in themselves are unique and have the potential for licensing for other medical and cosmetic uses. We believe the manufacturing and delivery assets are very important to the company as you need to be able to demonstrate scalability and commercial delivery.

CTB: What is the company’s technology?

DH: RCH-01 is our treatment for pattern baldness. This technology specifically focuses on replacing a deficit of healthy active dermal sheath cup cells (DSCs). Our product thesis is that these DSC cells control and maintain the population of dermal papillae (DP) cells in a hair follicle and the number of DPs determine the length and thickness of the hair fibre. In patients with pattern baldness (men and women), DSCs are compromised by the androgen hormone for no known reason. It is kind of like the reverse of unwanted or aggressive hair growth. Some people have five-o-clock shadows when they are 15 and others don’t shave until they are 20+. In pattern baldness, the androgen hormone compromises hair growth. RCH-01 addresses the deficit of the active DSC cells in the areas of pattern baldness in the scalp.

The RCT family of products* are for the treatment of chronic tendinosis including Achilles, patellar and both golfer’s and tennis elbow. In each case we are addressing a deficit of healthy active fibroblasts. The scientific thesis is that this chronic disease is the result of incomplete healing cycles due to a deficit of healthy functioning fibroblasts. Our early human clinical work on chronic tendinosis was done by our collaborator, Dr. David Connell, using fibroblasts isolated from the dermis. Our program is based on using fibroblasts isolated from the hair follicle due to their ability to express higher levels of type I collagen than dermal fibroblasts. Type I collagen is the main cell constituent of a tendon. (*RCT-A-01, RCT-P-01, RCT-G-01 and RCT-T-01)

RCS-01 is focused on replacing damaged or non-functioning fibroblasts in the dermis. Fibroblasts damaged by UV irradiation, smoking and other factors stop producing healthy amounts of collagen and the constituents of the skin’s extracellular matrix. As a result, the skin loses its texture. Similarly, acne and burn scars are targets for us to deliver highly expressive fibroblasts to initiate remodelling of the damaged skin.

The supporting manufacturing technology is also a critical asset to the company as we believe our process is scalable.

In addition, we have developed a very unique injection cell injection device for dermal injections for both our derm and pattern baldness program. This device minimizes shear force, optimizes staged cell delivery, and has a built in freezing element that obviates the need for any kind of anesthetic and can control both volume and depth of injection.

CTB: Where are you doing your trials? Why Europe and Canada?

DH: All of our trials are being conducted under the terms of reference from the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). Each trial is designed to meet these international standards of which all of the western nations are members. As such, all of our data up to Phase 2 are admissible towards Phase 3 pivotal programs in each jurisdiction.

Our RCS-01 trial is being done in Germany. The co-founder of the Company Dr. Rolf Hoffmann, is located in Germany and he is leading this initiative. Similarly, our RCH-1 treatment is being conducted in Germany at the Charité Hospital in Berlin. The Charité is one of the largest teaching and research hospitals in Europe. In addition, the technology itself was first developed in Dr. Hoffmann and Dr. McElwee’ s lab at the University of Marburg in Germany. There is history with these technologies in Germany and in addition, the cells are being processed by our contract manufacturer in Austria.

The clinical trial for RCT-A-01 will be conducted at the University of British Columbia because it is a world centre of excellence for sports medicine research including tendinosis. We have three very special advisers leading the development of the clinical trial protocol including Dr. Ross Davidson, Dr. Jack Taunton and Dr. David Connell. Each of them are key opinion leaders in sports medicine. It is also convenient to have this trial in our own city, but it was not the deciding factor. Reputation, skills and the ability to recruit patients were the deciding factors. The cells will be processed in Austria; however, we are moving to bring online a second contract facility validated in North America at the Centre for Commercialization for Regenerative Medicine in Toronto.

CTB: What is the strategy for the U.S. market?

DH: The US market will be addressed from two angles. First, we are presenting our fibroblast program to the US military under their peer reviewed medical research program. The DoD has a great interest in musculoskeletal research to deal with damaged tendons and skin. We believe our programs are ideally suited for their needs and are preparing filings for funding for clinical programs both in Canada and the US. The second approach is to undertake phase 2 clinical work on other target tendons in the US after we have gotten our RCT-A-01 program launched. Importantly, all of our trials are being conducted under ICH standards with the US regulatory pathways in mind.

CTB: Tell us about the deal you did recently with Shiseido and what your drivers were for that kind of deal?

DH: In 2013 we completed a geographic license with the Shiseido Company. Shiseido has its own hair research lab and they had come to the conclusion that our approach was the leading cell therapy technology for treating pattern baldness.

We were interested in doing a geographic license for Japan and parts of Asia for three main reasons. The first was to have a third party validation of our science and technology which is always important to a start-up company. The second was to be partnered with a large company whom had the resources (both human and financial) to conduct their own clinical program which along with our program would more than double the data being collected as we work towards establishing our dosing and other protocols for the technology in humans. Thirdly, Shiseido has committed to cell therapy in a significant manner including having commissioned and now opened a purpose-built cell processing centre in Kobe Japan, in the heart of the country’s regenerative medicine initiative.

Our ongoing collaboration will see technology improvements shared between parties. Financially, we received $4 million up front, future milestones totaling approximately $30 million as well as middle single digit royalties on sales. We are very pleased with our collaboration with Shiseido. They are a focused and dependable collaborator.

CTB: From our perspective, the company is significantly under-exposed both in the regenerative medicine industry and investment communities. Can you give us a sense of why you think that might be and your plans in that regard?

DH: Every company has its own chronological pathway to develop a profile in its industry and in the public domain. During the later stages of 2012 and through 2013 RepliCel was very concentrated on completing the Shiseido transaction as well as completing the filing of PCT patent applications on its fibroblast platform. Until we had completed the task of protecting our expanded technology, it did not serve the company to disclose these activities.

Now in 2014, we are completing the next round of financing, pushing hard on our clinical filings to launch 3 clinical trials and now have the freedom to discuss our programs. As such, we are going to be out in the public domain at conferences and doing roadshows in order to get the RepliCel story known and followed both in industry and in the capital markets.

CTB: While many of the companies in the cell therapy sector are focused on indications like cardiovascular disease, oncology, autoimmune disorders, or diseases of the central nervous system, RepliCel is going after sports injuries, baldness, and wrinkles. Do you see these indications as low-hanging fruit? Do these indications battle problems of lower-margin and/or reimbursement challenges?

DH: I would agree that our programs are low hanging fruit in the regenerative medicine business and that is an attractive distinction. Furthermore, as an autologous therapy, there is less risk of rejection than an allogeneic therapy.

Our therapies are designed to simply multiply a patient’s particular cells that are in deficit and then deliver it to the area of deficit. We are not differentiating or inducing cell change, we are simply letting the cells do what they normally do when placed in the area of the wound or damage.

A lot of regenerative medicine is focused on very complex processes that take a source cell and then engage in extensive cell manipulation and/or differentiation. While these treatments are going to arrive in the future, today they are very difficult therapies to perfect both in assuring that the cell differentiations are exact and controlled and that the cells are delivered in volume and stay and function where they are needed. Our process is just simpler. So, yes, this is low hanging fruit in terms of safety risk and the commercial development timeline.

In terms of margins, we are quite confident in our ability to demonstrate manufacturing scalability. In terms of reimbursement, initially we have only one product that would need pharmacoeconomic validation and that is the tendon program. However, we know that we can replicate and deliver a tendon program at a reasonable cost and that there is no current clinically established solution for chronic tendinosis. Therefore, we do not anticipate any problem pricing at a good margin. In terms of the derm program, the initial focus is a cosmetic therapy and I think pricing a solution for sun damaged skin, wrinkles, etc. should not be an issue. A treatment with good efficacy will have no problem getting a premium price. In terms of margins on a treatment for pattern baldness, it would be priced against the current standard of care which is hair transplant surgery. And at that price, we can make good margins.

CTB: Many of the cell therapy products already approved are in the musculoskeletal, dermatology, and wound repair space and none are a run-away success. Does it worry you to be also focused on these areas for your near-term opportunities?

DH: That is a statement on the relative efficacy and difficulty of delivery. Approval does not necessarily equate to commercial success. We believe that our programs will have significant efficacy for their targeted indications, be easily delivered and they will become the gold standards. But that is the nature of product development; you have go out and demonstrate efficacy! One comment on wound repair is that we are not at this time interested in pursuing open wound repair as that space has many solutions ranging from complex cell therapies to medical devices. It is a crowded space and we prefer at this time to prove our platforms where we are confident in our success and are not populated by other treatments.

CTB: Your initial focus is on autologous applications of the hair follicle-derived cells, what are plans to address some of the commercial (cost) challenges presented by autologous cell therapies already on the market?

DH: As mentioned above, we are focused in parallel to our clinical development programs, on developing a scalable process for our cell manufacturing. We see this as a process engineering program and not a development process requiring scientific breakthroughs. This development program is underway. It is also true that we believe that our hair follicle derived cells are immune privileged and that in the future, our technologies could well become an allogeneic offering. But that is down the road.

CTB: Few companies of ReplCel’s size and market cap are simultaneously tackling multiple products in clinical trials let alone also the concurrent development of a proprietary delivery device. Some might argue it would be more prudent to preserve capital to ensure you have what it takes to move one product further down the line. Can you give us a sense of your why you prefer this multi-pronged strategy?

DH: Our programs are inherently cheaper to develop and test than more complex cell therapies and for that matter, new chemical entities or biologics. We are simply expanding a patient's cells and giving them back to them. It is just cheaper to do than other therapies. As such, we can undertake more programs and I would argue that for shareholders, more shots on goal helps to mitigate risks associated with clinical programs. We believe we have a single focus on leveraging our core expertise around cells derived from the hair follicle organ and finding meaningful clinical applications for those cells. Optimizing the manufacturing and delivery of those cells just makes sense as part of the risk mitigation and value optimization of those assets.

CTB: To those investors interested in the regenerative medicine and cell therapy sector, where does RepliCel fit in the industry and how do you think it stacks up against sector comparables as an investment opportunity?

DH: I would characterize RepliCel as a mid-stage development company that exists between earlier approved products like Apligraf and Dermagraft for wound healing, and much more complex cell therapy clinical development programs. There are other mid-stage programs like Athersys' MultiStem and Mesoblast’s Mesenchymal Precursor Cells. However, I would argue that our simple cell replication process of a target cell deficit is simpler to manufacture and deliver than many cell therapies. So, I would characterize our development programs as lower risk. Nevertheless, I would also say to investors that regenerative medicine is going to be a huge field in the future as science and technologies evolve. This industry will be delivering incredible medical solutions for patients in the future.

CTB: Talk to us a little about the stock. It’s very lightly traded on both the Toronto venture exchange and over-the-counter bulletin board. What’s the plan going forward in terms of its listing and liquidity?

DH: Yes, it is true our trading has been low. That is a direct function of our tightly held stock, (management, founders, etc., own approximately 45% of the stock) and the fact that we were very focused in 2103 on our partnership and the patent filings. As discussed above, we are now launching our story on a focused basis that will see the company out on road shows delivering its message to potential investors. We think that RepliCel is an attractive and unknown story which will have a good reception. That effort is focused on addressing the valuation and liquidity. In terms of listing, the natural progression as we develop assets and valuation is to up list which we will be pursuing.

CTB: Where do you see RepliCel in 18-24 months?

DH: On a clinical basis, we will have data in both our tendon and derm programs and will be closing in on the clinical trial data for our treatment for pattern baldness. We should at that time be initiating at least one other tendon program and a phase 2 derm program. We will have developed clinical protocols for two new indications for our fibroblast platform (not yet being disclosed due to patent work) and we will have demonstrated scalability of our manufacturing platform. If we accomplish this as planned, we will have established an extremely valuable group of assets.

CTB: Thank you, David, for the opportunity to spend some time learning more about RepliCel and your plans for the company.

BioKorea 2014 - A focus on cell therapy and regenerative medicine

noreply@blogger.com (Lee Buckler) — Wed, 21 May 2014 00:13:00 +0000

I am pleased to be working this year with BioKorea and the Korean Global Stem Cell and Regenerative Medicine Acceleration Center (GSRAC) to host a full-day regenerative medicine track during the BioKorea conference at the end of May.

The Korean Ministry of Health and Welfare and regenerative medicine industry are blazing an innovative trail in terms of the commercialization of cell-based therapies which the world is watching with increasing interest. The Korean approach to and pace of product approvals has attracted the attention of investors, entrepreneurs, and regulators alike. On the Korean side, companies with commercial products are eager to expand into other markets. This involves technology transfer, securing partnerships, and subjecting their products to different types of regulatory requirements and clinical testing.

Companies outside of Korea are increasingly interested in identifying and how to involve Korea in their product development plans if it means an expedited pathway to commercial approval. This would likely involve identifying a Korean partner (or investors) and establishing a deep understanding of and relationships with the Korean regulators and industry.

In order to satisfy the needs of the industry both within and outside Korea it is critical to establish a closer working relationship between industry organizations, facilitate a better mutual understanding of the different markets, and create mechanisms for companies to further their goals by helping them identify partners, understand regulatory differences, establish critical relationships, find investors, etc.

This event is intended to create the initial framework for enhanced international collaboration between Korean and the global regenerative medicine industries.

Attendees will:

gain a more in-depth understanding of the opportunity and challenges in transferring a commercial product from Korea to other markets
hear case studies of collaborations between pharma and regenerative medicine companies
learn about the unique advantages and challenges of commercializing regenerative medicine products in Korea
learn about the perspectives of the pharma industry on regenerative medicine including cell-based therapies
have opportunity to establish contacts with international pharma executives, regenerative medicine companies, and potential partners and investors
have opportunity to identify companies from other markets potentially interested in (a) finding a partner, and/or (b) seeking to license and/or partner with Korean companies to take their product(s) into other markets
participate in networking activities with representatives of the global and Korean regenerative medicine industry
have opportunity to showcase the Korean biotechnology industry and capabilities to members of the global industry

The focus of the session is to facilitate increased collaboration between Korean and international regenerative medicine companies by creating a forum for exploring a better understanding of the Korean regenerative medicine industry and the potential for creating partnership, licensing, and investment opportunities emerging out of and interested in going into Korea.

We are pleased to have a full day of exciting content from both Korean and international speakers from multinational pharmaceutical companies, Korean representatives, and regenerative medicine companies from around the world.

In addition to the full-day program, we are also facilitating partnership discussions, networking events, and meetings (if requested with the Korean Ministry of Health & Welfare (formerly the KFDA).

BioKorea will be held May 28-30 at the KINTEX Exhibition Center in Goyang city near both Seoul and the Incheon International Airport. BioKorea, is organized by the Korea Health Industry Development Institute, KHIDI, as a government (Ministry of Health and Welfare)-affiliated organization and Chungcheongbuk-do Province, which is the center of Korean biotechnology, life sciences, and healthcare. The annual event first started in 2006 and is expected this year to attract 13,000 participants and over 260 exhibiting companies.

Track 3. Stem Cell and Regenerative Medicine: Thursday, May 29

This track aims at facilitating international cooperation and global commercialization

among companies in the stem cell and regenerative medicine, and will serve as a forum of discussion and deliberation for domestic and foreign business enterprises.

The applicants will have opportunities to take part in the following:

Session I: Introduction to the current status of domestic and foreign markets

and industries of stem cell and regenerative medicine

Session II: Case studies on technology licensing out between regenerative

medicine companies and large-scale companies

Session III: Publicity for domestic and foreign regenerative medicine companies

and their products

Partnering: For those companies which seek opportunities, a meeting place will be provided for their matching commercialization needs.

To help give a little more flavor to this event and the state of the industry in Korea, I had the opportunity to interview Professor Bryan Choi, who is Vice-Director of the GSRAC and has been much of the energy behind making the event a reality.

Welcome, Bryan. Can you start by telling us a little bit about the Global Stem Cell and Regenerative Medicine Acceleration Center (GSRAC) and how you yourself got involved in regenerative medicine?

GSRAC is a government commissioned strategic center that helps the Korean government (a) establish appropriate policy and regulations, (b) facilitate the translation of research to the clinic, and (c) assist RM companies accelerate market entry of their products. I, myself, am a researcher in regenerative and happen also to be involved in the management of the GSRAC.

Give us a sense of the state of the regenerative medicine sector in Korea.

RM is one of hottest areas of interest in the biomedical and pharmaceutical field and is being supported a lot by the Korean government. At the same time, we are also struggling to move forward with some of the technical and commercial aspects of these products.

How many cell therapy products are commercially approved in Korea to-date and how many are in pivotal trials?

I know approximately 18 cell therapy products have been launched in the Korean market and 3 of them are stem cell products.

In the past couple years, the world has been watching the development and commercialization of regenerative medicines, especially cell-based therapies, in the Republic of Korea with considerable and growing interest. This is largely driven by a number of cell therapies (including stem cell products) approved for commercial distribution by the Korean Ministry of Food and Drug Safety (MFDS) (formerly the KFDA) and the perception that the regulatory pathway to commercial approval appears to be more proactive and expedited than what exists in the United States and Europe. Can you comment on this?

I cannot say it is exactly proactive but it could be fairly perceived as relatively ‘expedited’ (compared to many other jurisdictions) due to the fast but not abbreviated process of MFDS. MFDS has similar principles to those of the US and Europe for the approval of cell therapy products. A final decision for a submitted product is case dependent.

We hear rumor that there are changes being considered to the regulation of cell therapies in Korea, can you comment?

We are currently talking about the need for a new, revised regulation that fits better for RM products but it is not yet formalized.

What is the level of interest or activity among Korean investors or large life science companies in investing in Korean regenerative medicine? Are you seeing any growth of interest in entering the Korean market from regenerative medicine companies outside Korea?

Recently they are getting more interested in RM. Some investment deals have been done recently between RM companies and Korean pharmaceutical companies. Where we have yet to see any notable activity is that of foreign investment or interest in the Korean RM market – even by the large multinational firms.

Tell us about BioKorea and your plans for a regenerative medicine track at this year’s conference. What do you hope to accomplish with this event?

In BioKorea 2014, we have a RM track (Track 3) which deals with business development and partnering in the RM industry. We invited more than 10 speakers of RM experts or from RM companies to talk about business models in regenerative medicine and partnering opportunities particularly between Korean and foreign RM companies.

How can people find out more about the event and what are the ways they can participate?

Please refer to the following websites: www.biokorea.org and www.gsrac.org.

Will representatives of the Korean Ministry of Food and Drug Safety (MFDS) be at the conference and available for participants to interact with if they wish?

MFDS representatives may be at the conference but we are also planning a separate meeting with MFDS for those interested.

Will there be opportunity for companies from outside Korea to identify and speak with potential Korean partners that might be potentially interested in in-licensing or out-licensing products?

It is always welcomed. You can participate in the business partnering any time before the end of BioKorea 2014, and may also give an introduction of your company in the session III of the RM track, if a slot is available. Please contact us (bryan [at]gsrac [dot] org) for the availability.

Thanks Bryan. I am very much looking forward to the event.

An Interview with BioCision. Why Innovating for Reproducibility is Good Business.

noreply@blogger.com (Lee Buckler) — Tue, 15 Apr 2014 15:00:00 +0000

BioCision is one of those companies that easily catches your attention (they use lots of bright colors) but might take awhile to be a regular part of your consciousness. After all, laboratory and bioprocessing tools are not the sexiest product category. Because of that, I tried to ignore BioCision for some time as a company that looked interesting but was too 'laboratory-focused' for my tastes.

What eventually forced me to take a closer look at this company was their clear commitment to two things: (1) making standardization and simplicity major driving forces for their innovation, and (2) online audience engagement not just for marketing but as an incredibly useful tool in driving the innovation feedback loop.

Also, I'll admit I was completely wrong about them only offering research laboratory tools. It turns out their applications may indeed offer even more value to those involved in clinical production.

They are a private company quietly but busily innovating around some of the most basic biologic handling issues in the lab or manufacturing suite -- often solving problems you didn't know you had until you are presented with the solution. They are an interesting mix of conservative yet innovative - bold but basic.

Long-story short: this company has really grown on me as the 'read deal' such that theirs is the first corporate advisory board I recently agreed to join (there's my disclosure).

A company is, of course, about a lot of people doing a lot of important things (big and small) that collectively make it a success but there's no doubt that Rolf Ehrhardt, the company's President and CEO, has stamped the company with his imprint. Because it is so clear to me that the BioCision culture and modus operandi is very much an imprint of Rolf's personality (as is often the case with companies a la Apple, Miltenyi, etc), I had to sit him down for an interview. That is what follows and I hope you find it interesting if not informative.

CTB: Tell us about BioCision and the sector(s) you serve?

The reproducibility of scientific studies has become a major issue in life science research from drug discovery and development through to clinical trials as researchers around the world continue to use different protocols for processes associated with sample preparation, cryopreservation and cold chain management. The scientific community is now becoming more aware of factors that affect sample integrity and experimental variability. By standardizing handling, storage, and transportation protocols we can vastly improve the quality and reproducibility of preclinical and clinical data, helping to accelerate the transition from lab research to drug development and market launch.

That’s where BioCision comes into play. In 2007 a group of scientists, biomedical engineers, and drug discovery experts founded BioCision to develop laboratory tools with standardization in mind in order to set a new bar in temperature control for biomedical samples. Today our products are used by researchers around the world in pharmaceuticals, biotechnology, academics and healthcare. We are positioned for continued growth in multiple sectors including cell therapy, regenerative medicine, biobanking, bioprocessing and cold chain management.

CTB: What would you say is BioCision’s underlying philosophy or business approach?

Our goal is to enable researchers to achieve the highest attainable levels of pre-analytical sample reproducibility and consistency – experiment to experiment, lab to lab and site to site. In order to accomplish this, we are very focused on our role as a leading advocate for the standardization of biomedical samples for basic, pre-clinical and clinical research.

In addition to our novel products, our business strategy is based on our ability to engage and mobilize the scientific community around this effort to improve reproducibility and develop new sample handling standards. We just announced the creation of a new scientific advisory board that includes internationally recognized researchers who are uniquely qualified to help BioCision develop additional products and support solutions that are able to address standardization challenges for our customers.

We look forward to working closely with the members of our advisory board, especially as we continue to expand our position as a global partner to support research and product development, in cell therapy in the years ahead.

We have also been able to leverage social media platforms not only to share and support our message of preserving sample integrity but engaging our audience on their innovation needs. For example, we have worked hard to develop and maintain a blog called “Sampling Science®” to keep our readers informed about standardization efforts in cell therapy research and other fields of interest. Most recently, we created a discussion group called “Scientists Against Sample Abuse™” - or “SASA™” - to continue this important conversation about improved sample handling with customers and fellow advocates.

In the end, these efforts have resulted in a growing community of laboratory researchers and clinicians who understand the significant impact that sample/drug handling and storage techniques can have on study results and even the efficacy of new biological therapies.

CTB: Your products seem to have a very strong visual design concept that’s reminiscent of Apple or Miltenyi. How important is that to the company?

The design of our products is positioned as a significant value-added benefit for our customers. On the surface, the fluorescent colors we use for our products bring a spot of fun to the laboratory and help us stand out at exhibitions and medical conferences. But there’s much more behind our design strategy.

The use of color-coding also helps laboratories work smarter and reduce the risk of errors. Most labs still use buckets of ice, refrigerators, water baths and mostly white, grey or glass containers for biomedical sample handling and storage. This makes it difficult to organize samples in an intuitive way, leading to delays or errors in processing that can affect sample quality and results.

We try to design products based on how scientists actually interact with samples. We make it possible for researchers to use color to streamline operations and improve the quality and efficiency of lab procedures. The use of color makes it easier to store and organize similar samples. Labs can develop storage protocols based on color so any researcher is able to easily find a sample. We envision a future where products can be custom-designed and color-coded to meet the needs of major laboratory systems around the world.

CTB: Has the company done much in the way of partnerships with larger companies?

Last year, we announced an agreement with the American Type Culture Collection (ATCC), the world's leading provider of authenticated biological material, that enables the development a custom line of products including the CoolCell LX cell cryopreservation device. As a result, ATCC cell-freezing protocols recommend the use of CoolCell LX containers to maintain an optimal controlled and standardized freezing rate. Their protocols are widely used as the industry standard in global biomaterials research.

This year we are working on a couple of exciting projects. The first is with TxCell, a French company that is developing cell-based immunotherapies for the treatment of severe chronic inflammatory diseases. Several of our products are being used to support temperature control and the processing and handling of cell therapies in their Phase IIb clinical trial investigating the use of an antigen-specific regulatory T (Ag-Treg) autologous cell-based immunotherapy for the treatment of patients with moderate to severe refractory Crohn’s disease. In this effort our CoolCell alcohol-free cell freezing container is being used to freeze Type 1 regulatory T (Tr1) cells in Aseptic Technology ampules.

It’s an interesting case study, because in previous trials, TxCell used electronic programmable freezers to manage the cell freezing temperature. However, the Phase IIb trial will involve collection of cells from multiple sites, prompting TxCell to choose the CoolCell containers for cell freezing, because they provide the same freezing outcomes as programmable freezers, while being much easier and much less expensive to deploy to multiple clinical sites due to their portability, lack of necessary maintenance and ease of use. TxCell is also using our CoolRack and CoolBox ice-free cooling systems in the cell handling stage to standardize the temperature of the cell preparation, media, and cryopreservatives to 4°C without the use of ice and reduce the risk of drug-product contamination. Because the efficacy of regulatory T cells can change without proper and reproducible handling methods, the standardization of pre-analytical drug sample handling, cryopreservation and storage are key to ensuring consistent trial results.

TxCell will present additional details on the use of CoolCell containers for cell freezing during a poster session at the 20th Annual Meeting of the International Society of Cellular Therapy in Paris this April.

A related paper has also been published in the March issue of BioProcess International titled “Effective Cryopreservation and Recovery of Human Regulatory T Cells.”

Earlier this year, we received a $4 million equity investment from Brooks Automation, a global provider of automated sample storage systems for compound management and biorepositories. The investment initiates a strategic partnership that will support the development of innovative sample handling solutions for cold chain management and the handling of drug products.

In just the past few years, numerous independent publications have endorsed and recommended the use of BioCision products in their methods and protocols sections. We look forward to partnering with more companies in the years ahead by offering innovative solutions to temperature control and sample handling and storage needs.

CTB: What product offerings do you have in the cell therapy space?

We are perhaps best known for our CoolCell cell freezing containers, which provide a reproducible freezing rate of 1°C/minute when placed in a -80°C freezer and have become the new industry standard for cell cryopreservation in research and clinical applications, including cell therapy. The rate at which stem cells are frozen is crucial to the overall success of cryopreservation process and the freezing rate should be measurable in a way that does not rely on the freezer size or model and should also be equal across all samples in a freezer. Our CoolCell SV series is specially designed to fit closed-system, injectable glass ampoules that are popular in the cell therapy space where aseptic vials are critical for minimizing contamination.

A recent independent example of our product in use in the cell therapy space was published in the Journal of Autoimmunity. Researchers cited the use of a CoolCell freezing container for studying the role of T cells in myasthenia gravis. The CoolCell freezing container was used to gradually freeze cells isolated from the blood of both patients and healthy donors for long-term storage while sample collection was underway.

Our CoolRack® and CoolBox™ ice-free cooling systems are also commonly used to standardize the temperature of cells, cell media, and cryopreservatives prior to the cryopreservation stage.

More recently, we have developed a product called CoolStation™ which is a mobile ultra-low temperature workstation. The CoolStation systems provide an ultra-low temperature open-top work space through the use of dry ice or LN2 where a variety of operations such as cryogenic packaging, sample sorting or transport, or sample freezing can be performed. For example, by placing a CoolStation next to a biosafety cabinet during cell preparations, cells could be placed into a CoolCell freezing container and the CoolCell could be directly placed in the CoolStation for immediate freezing, limiting the non-frozen exposure to DMSO or other potentially harmful cryoprotectants.

CTB: Why have you decided to expand into the field of cell therapy?

Cell therapy is a rapidly expanding field that holds great promise for future clinical applications and early trials continue to yield positive results. But in the grand scheme of things this is still a very new field of research. Just a few years ago it was often assumed that small variations in cell temperature or cryopreservation would not dramatically affect the outcome of an experiment. As cell therapy research has advanced we now have a much better understanding of how stem cells behave in different environments, and it’s clear that even slight changes in temperature management can have major implications for the efficacy of the final therapeutic product. Today, we regularly find and share reports on the importance of proper sample handling, particularly related to temperature control, to ensure the reproducibility of cell therapy research.

For example, following workshops organized by the U.S. National Institutes of Health (NIH), the journal Nature recently launched an initiative to more systematically ensure key methodologies are reported by researchers, including the creation of a checklist prompting authors to disclose certain technical and statistical information.

The movement to minimize irreproducibility in cell therapy research has led to growing interest in BioCision’s innovative solutions as researchers discuss different tools and protocols for handling and storing samples.

CTB: How do you decide on new products to develop?

We remain focused on sample standardization in all areas of life science and clinical research and are continuously developing and improving tools that address common problems related to biomedical sample handling and storage.

Currently we are working on three main areas of product development. First, we continue to advance our cell freezing and related workflow solutions, with a special focus on stem cells and primary cells for research. Second, we are developing new solutions for sample handling before and after long-term storage (i.e. Biobanking). And third, we are studying clinical and diagnostic applications where maintaining consistent temperatures - either cold or warm - is critical, for example in home-based assays and cold chain management for short transport.

In these efforts we are always actively seeking partners and collaborators who have an application for our innovative technology.

CTB: Are you working on any new technologies that the cell therapy sector should be excited about?

Yes. We have a number of products related to the cell cryopreservation workflow in various stages of development, some of which will be released at the ISSCR annual meeting in Vancouver in June, and these products will directly relate to cell therapy and aid in further standardizing cell handling. And, as mentioned above, we are working with Brooks Automation to develop new products that will also apply to the cell therapy workflows.

CTB: Will you be at the upcoming ISCT conference?

Yes, we’ve been looking forward to this year’s conference for quite some time. Working closely with TXCell, we are excited to present an abstract during the poster session that will analyze the benefits of using our CoolCell freezing containers in the manufacturing of an autologous antigen-specific Treg (Ag-Treg) cell-based immunotherapy for the treatment of patients with severe refractory Crohn’s disease. Researchers investigated CoolCell freezing containers as an alternative to the classical programmable freezer and developed a new standardized method of cell therapy product cryopreservation. It’s a major first step towards the use of cell therapy in the clinic.

We also plan to present an abstract in collaboration with researchers from Dr. Jeff Bluestone’s lab at the University of California, San Francisco who are investigating the cryopreservation of peripheral blood mononuclear cells (PBMCs) with the intention of isolating regulatory T lymphocytes (Tregs) for autologous cell-based immunotherapy treatment in patients with autoimmune diseases or transplantation recipients. Similar to the TxCell study, the UCSF team investigated CoolCell freezing containers as an alternative to programmable freezers to cryopreserve PBMCs. They concluded CoolCell containers can help overcome challenges associated with multiple clinical site collections in cell therapy research because our device is less expensive, portable, lacks necessary maintenance, and is easy to use.

CTB: Thanks for the taking this time with us. We look forward to hearing more about what the company has in store for the cell therapy sector soon!

Oh ya. And remember that bit about social media? Check them out:

https://www.facebook.com/BioCisionLLC https://twitter.com/biocision

http://blog.biocision.com/

http://www.labtube.tv/labtubemeets.aspx?vid=159051

http://www.linkedin.com/company/1225223

http://www.linkedin.com/groups/Scientists-Against-Sample-Abuse-SASA-6597044

US vs Regenerative Sciences Comes to End

noreply@blogger.com (Lee Buckler) — Tue, 04 Feb 2014 16:45:00 +0000

A court case we blogged a fair amount about even before it started (see the first post in 2008) appears to have finally come to an end in the DC Circuit Court's decision to uphold the ruling in favor of the FDA.

United States Court of Appeals
FOR THE DISTRICT OF COLUMBIA CIRCUIT

Decided February 4, 2014

No. 12-5254

UNITED STATES OF AMERICA,
APPELLEE

v.

REGENERATIVE SCIENCES, LLC, A CORPORATION, ET AL.,
APPELLANTS

Appeal from the United States District Court
for the District of Columbia
(No. 1:10-cv-01327)

It has been an interesting ride. Chris Centeno and his partners (and investors) have been the lightning rod for a lobby and debate around the right way to regulate the use of cells therapeutically - particularly one's one cells. The arguments became more sophisticated as the case wore on and, as so often happens, the ground shifted considerably while the case was ongoing. The case and Chris Centeno's unparalleled commitment to the cause has contributed significantly to a debate that is important to have and I suspect is not over simply because the case is done.

Dr. Centeno and I have had our differences but now is the time to recognize his contributions to the debate and, more importantly, to the field. We look forward to his future contributions to developing robust, cell-based treatments for patients.

Regenerative Medicine / Cell Therapy at Biotech Showcase 2014

noreply@blogger.com (Lee Buckler) — Thu, 02 Jan 2014 17:33:00 +0000

As many of you know, during the week of January 13, healthcare and life science executives meet investors in San Francisco for a flurry of conferences, meetings, parties, and general mayhem.

The EBD Biotech Showcase is one of several events taking place that week and it the one of particular interest to us because it has the most concentrated focus on regenerative medicine and cell therapy companies. The full list of presenting companies is here. For those of you on Twitter, follow #BTS14.

We've gone through and picked out the regenerative medicine and cell therapy content.

Sponsored by the Alliance for Regenerative Medicine, the 4th annual Regenerative Medicine Industry Briefing kicks off the meeting at 8 am on Monday morning (Jan 13th) . This year it is in the big plenary room and it promises to be the best yet. Invite your friends, investors, partners, and media contacts.

We spotted the following 29 companies scheduled to present at various times as shown on the schedule spanning Monday through Wednesday:

Adaptimmune
Advanced Cell Technology
AlloCure
Bone Therapeutics
Cell Medica
Cell2B
CTI Clinical Trial and Consulting Services
Cytori Therapeutics
DiscGenics
Fibralign
Harvard Apparatus Regenerative Technology
International Stem Cell
Kiadis Pharma
MaxCyte
MolMed
NeoStem
Neuralstem
Northwest Biotherapeutics
NovaRx
PharmaCell
Pocastem
Q Therapeutics
RepliCel Life Sciences
RhinoCyte
Sernova
TiGenix
TVAX Biomedical
TxCell
ViaCyte

Because it is a full schedule with 4 concurrent tracks, we made our own schedule of regenerative medicine and cell therapy presentations. In case it is useful, we provide it here for you to download and use.

2014 EBD Biotech Showcase Regenerative Medicine and Cell Therapy Presentation Schedule (PDF)*

This schedule is sponsored by

* We can't promise we didn't miss a company, screw up the schedule somehow, or that EBD might not change things between now and the event. You should really check this against the EBD program and please... rely on this resource in your discretion.

An interview with GlaxoSmithKline's Dr. Jan Thirkettle

noreply@blogger.com (Lee Buckler) — Mon, 16 Dec 2013 12:00:00 +0000

An interview with:

Jan Thirkettle, BSc, PhD

Head, Advanced Therapy Delivery

GlaxoSmithKline Medicines Research Centre

In anticipation of Phacilitate's 2014 Cell and Gene Therapy Forum to be held at the end of January in Washington, DC, I invited the Phacilitate team to provide blog readers with an interview of one of their upcoming faculty. The result is the very interesting Q&A thread you see below about GSK's call and gene therapy program combined with some of his thoughts on the sector.

Phacilitate: You head the Advanced Therapy Delivery group at GSK – please could you outline your specific goals and activities as an organisation for us? Which projects/technologies are you prioritising currently, and why?

Dr. Thirkettle: Our immediate priority is to deliver the Chemistry, Manufacturing and Controls (CMC) aspects of a collaboration we already have for an investigational ex vivo gene therapy currently in late stage development with TIGET, the San Raffaele Institute for Gene Therapy in Milan.

Through this collaboration we believe that we have a responsibility to complete the process for development of gene therapy, get it to file and make it available to more patients. We are really excited by the clinical data which TIGET have generated on the current programs thus far, but this is also laying really important groundwork for how we will work on other products in that collaboration and giving us real insight into the issues which we need to tackle in order to be able to progress following opportunities in this collaboration and make these autologous therapies more scalable.

This feeds into our second priority, which is to develop more efficient manufacturing technologies that can allow us to reduce the cost of goods, reduce the complexity, and increase the scalability of ex vivo gene therapy products. This is important within our existing collaboration but also it is going to be critical to allow us to engage with other opportunities which might have larger patient numbers.

Thirdly, whilst the immediate focus of our group is on the CMC aspects (process development, vectorology, cell characterisation, QC), we are also very focussed on working across the GSK platform organisation to leverage the expertise we have from our pharma and vaccine background and which is relevant to this area. This engagement and strategic planning is also critical to make sure that we build expertise in all of the other really key functions; for instance, this is an area where really strong understanding of the regulatory and the quality aspects is absolutely critical.

Phacilitate: This January, you will be joining a panel of big pharma representatives tasked with answering the questions of whether 2014 is the year pharma get serious about Cell & Gene Therapy, and whether it has been a lack of innovation or a lack of proven products that has held them back in the past - what’s your initial response to these questions?

Dr Thirkettle: I’m not sure it’s fair to say that big pharma has yet to get serious, if you look at the investments and deals of the past 3 years. I certainly don’t see any lack of innovation out there, it is almost the opposite. We talk about cell and gene therapy as a modality, but it is a huge, broad field; as broad as ‘biopharmaceuticals’ which is a field with many approaches and technologies.

Within the cell and gene therapy field we have a very large number of modalities, different cell types, different approaches, and a huge diversity of technologies. And they are all evolving at a really blistering pace. So one of the challenges for anybody who is outside of the groups who are really driving that core science is to know which is the right bit to grab hold of and it’s not the sort of area that you can just dabble in if you want to be really be effective and add value.

Rather than lack of innovation, then, the challenge is maybe more of knowing how to pick the winners at the right time and understand the potential utility of that technology/product at this early stage. I think the key thing is that it requires a number of elements to come together; you need the biology and the underpinning science to be strong; you need a therapy with good clinical data to really evidence that and discharge some of the risk; and then you need an understanding of how broadly applicable that technology might be - is there some sort of platform that sits behind it that spreads your risk? And finally, you need an understanding if whether you can actually make that product, can you scale it up?

Phacilitate: You are also kindly chairing a session which explores the long-standing but still critical challenges in developing cost effective and robust manufacturing models for cell & gene therapies at industrial scale. What progress do you see being made in this regard? To what extent will it be a case of these problems being solved relatively quickly and easily as big pharma come on board more and more, as some have suggested?

Dr Thirkettle: There are some massive challenges, but I suppose I would never underestimate the ability of really committed scientists, whether from academia or biotechs or big pharma, to solve those problems given enough focus and time.

I think the key is creating a focus because in a way it goes back to the previous point - this is a hugely broad field. When you stand back and look at this mass of really exciting science and opportunity, the challenge actually is to understand where to focus that investment in time and money. It becomes an awful lot easier to create that focus and energy to invest where you have got really compelling clinical data - you know you have got something that can potentially be a product. Equally if you can have confidence that a technology has multi product utility, your ability to invest is that much greater. If you can make a strong case on either of those points then you’re in a good position to create the focus needed to drive investment in the manufacturing and commericalisation aspects.

Phacilitate: Finally, what are you hoping to get out your participation in the meeting this January?

Dr Thirkettle: I’m really hoping first of all to meet lots of other people who are facing the same challenges and learning the same things. I think it is really exciting to see the journey over the past few year with increasing discussions around “How do we make these therapies? ” rather than “Will these therapies ever work?” This is a field which would potentially be greatly enabled if there players could find a way to share learnings as much as possible about what works and what doesn’t. There is a huge opportunity to work in a pre-competitive mode. It is in everybody’s interest because there are so many challenges, so many things to learn. I think this is a forum which makes it really easy to have those conversations, and start to build those relationships that, ultimately, should help everyone.

Cell Therapy Industry Infographic

noreply@blogger.com (Lee Buckler) — Sat, 29 Jun 2013 00:56:00 +0000

Here is an infographic of the data in one of my recent presentations.

Enjoy. Consume. Share. Critique.

Commercialization of Regenerative Medicine: Learning from Spin-Outs

noreply@blogger.com (Lee Buckler) — Mon, 22 Apr 2013 01:51:00 +0000

The meeting “Commercialization of Your Regenerative Medicine Research: Lessons from Spin Out Successes” was hosted by the Oxbridge Biotech Roundtable (OBR) (Oxford, UK) at the University of Oxford in February, 2013, and attracted a multi-stakeholder audience spanning academia and industry.

The event featured case studies from Gregg Sando, CEO, Cell Medica (London, UK), John Sinden, CSO, Reneuron (Guilford, UK), and Paul Kemp, CEO and CSO, Intercytex (Manchester, UK).

OBR is a student-led initiative with over 7000 members across eight different UK and US locations with a mission to foster a conversation about the healthcare and life sciences industry.

Anna French and David A. Brindley, along with some of my assistance, captured and have now published the main themes of the meeting and the major questions facing the regenerative medicine industry and its rapidly emerging subsets of cellular and gene therapies.

Notably, we discuss the compatibility of regenerative therapies to the existing healthcare infrastructure, biomanufacturing challenges (including scalability and comparability), and the amenability of regenerative therapies to existing reimbursement and investment models. Furthermore, we reiterate key words of advice from seasoned industry leaders intended to accelerate the translation path from lab bench to the marketplace.

To read the review see: Commercialization of Regenerative Medicine: Learning from Spin-Outs

Anna French, R. Lee Buckler, and David A. Brindley. Rejuvenation Research. April 2013, 16(2): 164-170. doi:10.1089/rej.2013.1423.

2013 Annual Regenerative Medicine Industry Report

noreply@blogger.com (Lee Buckler) — Thu, 18 Apr 2013 04:58:00 +0000

The Alliance for Regenerative Medicine announced today the release of the 2013 annual regenerative medicine industry report. Here is the announcement in the Wall Street Journal online.

I'm proud to have been a part of putting it together and hope people find it useful. It is available for download on the ARM website here.

In addition to the complete download, ARM will make many of the figures, charts, tables and sections available for members to download and use in their own publications and presentations. Watch for these resources to be announced soon.