Image created by Google Gemini Pro

Patients diagnosed with degenerative eye disorders, such as retinitis pigmentosa (RP) or choroideremia, face a gradual loss of light-sensing cells that eventually leads to blindness. Historically, most treatments could only aim to slow disease progression; however, bioengineering is changing toolkits, allowing scientists and doctors to do more. Optogenetics is an excellent example of this, and it has already ushered in a paradigm shift: Clinicians are being empowered to aim for stem cell-free restoration of vision, rather than degenerative delay.

If this sounds familiar, maybe you remember my top pick in the Spring 2025 edition of Regenerative Medicine News Under the Microscope. I’ve also covered this story on my YouTube channel. However, there have since been both significant progress and shifts in the competitive landscape, with another major player emerging in the race to restore functional vision using optogenetics.

The tool

To recap: Optogenetic tools enable us to engineer cells and molecules so that aspects of their activity can be controlled using pulses of light. In the case of vision repair, a gene encoding a light-sensitive protein, called an opsin, is virally delivered directly into the neurons of the eye, which weren’t otherwise photosensing themselves. Because these neurons are already patched into the central nervous system (CNS), once they’re equipped with opsins, they can transmit light-related signals to the brain and reproduce aspects of vision.

Crucially, the described approach is “mutation-agnostic.” There are over 100 known genes and more than 1,000 corresponding mutations that can contribute to RP alone, meaning that traditional corrective or compensatory gene therapy would require a customized treatment for each genetic anomaly. Such an approach would represent greater cost and complexity. Optogenetics bypasses this problem entirely, making it a viable treatment for many forms of retinal blindness without requiring genetic testing per se.

Currently, two companies – Nanoscope Therapeutics and Ray Therapeutics – are leading the clinical translation of this technology. While both use optogenetics, their opsins of choice differ, as does their progress.

The frontrunner

Nanoscope Therapeutics (the team I’ve highlighted previously) is developing a therapy called MCO-010 (also known as vMCO-I in early trials, now branded as MOGENRY). MCO stands for multi-characteristic opsin (MCO), representative of their unique approach to the problem. Their opsin is a synthetic, broad-spectrum actuator that even works well in low-light conditions. It was designed to incorporate three different protein domains that each respond to a different wavelength of light (one for red, one for blue and one for green). Why is this a critical advance? Opsins typically respond to just a single wavelength, which is among the reasons why they are less sensitive and need more light to work. Previous attempts to use single-wavelength opsins to restore human vision have required wearable devices that amplify light inputs. While that work was pioneering and represented a significant milestone, the ability to see without external technological aid is the ideal outcome of new therapies. Nanoscope’s MCO is thus an exciting innovation in the field.

MCO-010 is delivered via a single, in-office intravitreal injection and targets the highly dense bipolar neurons in the retina. These cells are signalled by photoreceptors under healthy circumstances, but since the photoreceptors are degenerating in disease contexts, direct targeting of the second-line bipolar cells offers a solid workaround. Newly light-sensing bipolar cells can now transmit information to their neighbours (ganglion cells), which in turn signal the brain.

Nanoscope had previously completed a a Phase I/IIa open-label, dose-escalation study that evaluated two doses in 11 patients with advanced RP. Those data are published here. According to Nanoscope, the treatment was found to be safe and well-tolerated over five years of monitoring, with no serious adverse effects. They’ve also completed a Phase IIb/III RP study since I last reported on their progress, though those data have not yet been peer reviewed. The new study had 27 patients enrolled, according to the clinical trial record, despite initial intentions to look at just 18 subjects at two doses. Frankly, I thought it was amazing to see their patients (no pun intended) go from being completely unable to detect a designated light source before treatment, to being able to walk straight towards it post-treatment. The company currently considers its therapy to be registration-ready for RP and Phase III-ready for Stargardt disease (with their Phase II data for this indication published here).

A new player emerges

Another company looks to be a serious competitor in this space. Ray Therapeutics is now firmly in the race, with investors betting big on their efforts.

Ray’s lead candidate, RTx-015, utilizes ChRown opsins; these are improved channelrhodopsin variants, referred to as CoChRs. The team has bioengineered the protein to further increase its light sensitivity, though their approach was different than Nanoscope’s MCO, as ChRown still appears to be a single-wavelength opsin. Despite this, Ray reports that ambient lighting conditions are sufficient for activation without the use of amplifying goggles. Again, this offers a major advantage over original technologies in this space. I’ve not seen preclinical data published explicitly under the Ray Therapeutics banner, but they often cite this paper as foundational for their venture, and Dr. Zhuo-Hua Pan is listed both as an author on the paper and as an inventor on Ray Therapeutics’ major patent. Their data were also presented in a webinar through the Choroideremia Research Foundation’s series on YouTube. The talk was delivered by Dr. Raj Agrawal, Vice President, Clinical Development & Therapeutic Area Head.

Like MCO-010, RTx-015 is designed as a one-time intravitreal gene therapy that aims to provide lifelong benefits. Ray Therapeutics is currently running its Phase I trial, evaluating up to four dose cohorts of RTx-015 in approximately 18 patients with either RP or choroideremia. They’ve enrolled 10 patients so far. Instead of delivering their gene to bipolar neurons, however, they are targeting ganglion cells – the next link in the visual chain.

The scientific and regulatory community is showing strong support for Ray’s approach. Investors have rallied behind them, recently closing a US$125 million Series B financing round. Furthermore, the therapy has secured a Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), complementing its existing Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA).

Outlook

Optogenetics for vision restoration is a rapidly advancing field with several companies vying for leadership, but Nanoscope Therapeutics and Ray Therapeutics have emerged as the most compelling frontrunners in my view. Nanoscope leads the pack overall with statistically significant Phase IIb/III results and a Biologics License Application submitted to the FDA, making them the closest to regulatory approval relative to their competitors.

Furthermore, both teams have diversified their strategies. Ray Therapeutics is pursuing both ganglion cell (RTX-015) and bipolar cell (RTX-021, pre-clinical) targets, while Nanoscope Therapeutics is looking at both viral (MCO-010) and non-viral (MCO-020, pre-clinical) opsin delivery approaches.

Notably, GenSight was the scientific first-mover, widely considered a trailblazer in the field. However, their candidate requires the light-amplifying goggles that I mentioned earlier. This strategy probably won’t win out with patients (and therefore, investors). It will likely be adapt-or-fall behind for optogenetic tools relying on external devices.

There are currently other companies with similar offerings at various stages of clinical development, but none are as active or as advanced along their R&D pipelines just yet.

I’ve put together a table comparing the two leaders in this space:

Both Nanoscope Therapeutics and Ray Therapeutics represent a major leap forward in treating late-stage degenerative eye diseases. By utilizing mutation-agnostic optogenetics, these single-injection therapies avoid the complexities of traditional gene editing to restore functional vision.

Whether or not these technologies will eventually lead to full vision restoration remains an important open question in the field right now. How far will optogenetic tools take us? Will they ever be able to reproduce native vision as research and development continue? Time will tell.

I’m very excited to see how this landscape continues to change!

Michael May at Bio-Europe 2024. Image courtesy Felixfoto (Felix Buchele)

Fifteen years ago, regenerative medicine in Canada stood at an inflection point. The science was world-class, the academic talent was undeniable, and the promise of cell and gene therapies was beginning to capture global attention. Yet there was a critical problem: too many discoveries were trapped in laboratories, unable to navigate the long and complex journey to commercialization and patient care. Into that gap stepped the Centre for Commercialization of Regenerative Medicine (CCRM), an organization built not only to advance science, but to connect people, capital, infrastructure and ideas in ways that could transform an emerging industry.

The origins of CCRM are deeply tied to my story and that of CCRM’s co-founder, Dr. Peter Zandstra. This blog will share my motivations and inspirations for establishing CCRM.

I was raised in rural Ontario near the Grand River and close to where Alexander Graham Bell lived. I learned about Bell at an early age and was inspired by his blend of invention, experimentation and social impact. Bell’s telephone revolutionized communication, while his work with the hearing impaired reflected a commitment to improving lives through innovation. Decades later, BlackBerry’s development of the smartphone along the same river reinforced another lesson: transformational technologies often emerge where networks of talent, entrepreneurship and infrastructure converge.

While studying chemical engineering at the University of Toronto, I saw transformative research taking place. One thing that struck me was how slowly promising discoveries moved toward patients. That realization became a defining mission: To accelerate the translation of breakthrough science into viable therapies and sustainable companies.

With one company under my belt – Rimon Therapeutics – I was offered an exciting opportunity in 2010. MaRS Innovation (now TIAP), the Stem Cell Network, leading regenerative medicine researchers and the University of Toronto asked me to design a commercialization strategy for Canada’s emerging regenerative medicine sector.

Armed with $15 million in seed funding from the Government of Canada and a broad coalition of partners from academia, government, industry and investment, CCRM launched in 2011 with a mandate unlike traditional research organizations. Its role was not to fund science, but to build the ecosystem required to turn scientific promise into real-world impact.

Over the next decade and a half, CCRM has become one of the defining organizations in Canada’s regenerative medicine landscape. It operates at the intersection of science, business and investment, helping bridge the so-called “valley of death” that often prevents early-stage technologies from reaching commercialization. CCRM has supported hundreds of academic and industry projects, helped launch and scale dozens of companies, and contributed to attracting hundreds of millions of dollars in investment into the sector. CCRM’s role in gene therapy company AVROBIO and its 2019 initial public offering enabled CCRM to seed its for-profit investment arm, CCRM Enterprises.

Indeed, several of Canada’s most recognized regenerative medicine success stories carry CCRM’s fingerprints. BlueRock Therapeutics, now a subsidiary of Bayer AG, emerged as a global leader in cell therapies for neurological and cardiovascular diseases. Notch Therapeutics advanced innovative stem cell-derived immunotherapies before being acquired by Roche. Montreal-based ExCellThera has advanced technologies to expand blood stem cells for transplantation and other applications, generating best-in-class clinical outcomes. Together, these companies demonstrate that Canada can generate globally competitive regenerative medicine companies rooted in domestic science and talent.

Thanks to Peter Zandstra, CCRM recognized earlier than many others that manufacturing would determine whether cell and gene therapies could become scalable, accessible health care solutions. Unlike traditional pharmaceuticals, these therapies require highly specialized production processes involving living cells, viral vectors and sophisticated quality controls. Manufacturing complexity has become one of the industry’s greatest bottlenecks and a primary reason why therapies remain so expensive.

CCRM responded by investing heavily in biomanufacturing capabilities, process development and analytics. A momentous achievement was establishing OmniaBio Inc. in Hamilton, Ontario. As Canada’s largest cell and gene therapy contract development and manufacturing organization, OmniaBio represents a major strategic asset for Canada. It provides the infrastructure needed to manufacture therapies from preclinical stages through commercial production, helping ensure promising treatments can move beyond small clinical trials and toward widespread patient access.

The importance of accessibility cannot be overstated. While the global cell and gene therapy market is projected by multiple analysts to exceed US$200 billion within the next decade, there is a growing tension between innovation and affordability. Approved therapies today often cost hundreds of thousands or even millions of dollars per patient. Those price points create enormous challenges for health-care systems, and they raise difficult questions about sustainability and equity.

The future of regenerative medicine, therefore, will not simply be measured by scientific breakthroughs, but by whether therapies can become accessible to the patients who need them most. Not every disease or condition will require, or justify, an expensive, highly personalized cellular therapy. The industry is increasingly recognizing the significance of prioritization: Focusing advanced therapies where they can deliver transformational outcomes for patients with serious unmet medical needs, particularly in areas where conventional medicines fall short.

This is where CCRM’s next phase becomes especially critical. The organization is now evolving from a Canadian innovation hub into a global network model. Existing international hubs in Australia and Sweden demonstrate how CCRM’s ecosystem-building approach can be replicated and adapted in other regions to accelerate regenerative medicine development worldwide. Rather than operating in isolation, these hubs are designed to work collaboratively and synergistically, sharing expertise, infrastructure and investment opportunities across borders.

At the same time, CCRM is positioning itself at the forefront of transformative technologies that could reshape the future of the field. Through its collaboration with IonQ, CCRM has established DeepTech Bio Lab™, a centre of excellence focused on integrating quantum computing, artificial intelligence (AI) and emerging technologies to accelerate biotech companies, especially those in advanced therapies. The initiative reflects a broader recognition that biology is becoming an increasingly data-intensive and computationally complex field. AI-driven discovery tools, advanced modeling systems and quantum-enabled optimization may eventually help reduce development timelines, improve manufacturing efficiency and lower costs.

What has distinguished CCRM throughout its history is its understanding that innovation ecosystems matter as much as scientific discovery itself. Talent pipelines, manufacturing infrastructure, investment networks, public-private partnerships and global collaboration are all essential ingredients in building a sustainable regenerative medicine industry.

Cell and gene therapies have been proven to work. As CCRM enters its next chapter, the challenge now is scaling these therapies responsibly, affordably and globally. If you’d like to help, let’s connect.

Image: Pixabay

Humans have been obsessed with eternal youth for thousands of years. The search for elixirs, tonics, and the philosopher’s stone can be traced back to ancient civilizations. Only in the 1990s, however, did research show that lifespan may be modifiable. Pathways such as mTOR and insulin/IGF-1 were identified, and the field gained respect in academia. More recently, longevity research has exploded. The seminal paper, “The hallmarks of aging,” established nine common denominators of aging that appear during the aging process, accelerate aging if accentuated, and slow the aging process if improved.

It was updated to twelve hallmarks in 2023, which are listed below:

• genomic instability (DNA damage and mutations)
• telomere attrition (shortening of the protective caps at the end of chromosomes)
• epigenetic alterations (reversible DNA modifications that turn genes on and off, such as DNA methylation or histone modification)
• loss of proteostasis (accumulation of misfolded or damaged proteins)
• disabled macroautophagy (inability to remove damaged proteins, organelles, or cells)
• deregulated nutrient sensing (poor detection of energy and nutrient availability)
• mitochondrial dysfunction (inefficient energy production)
• cellular senescence (“zombie cells” that can’t divide anymore but secrete inflammatory signals)
• stem cell exhaustion (loss of ability to repair and regenerate tissue)
• altered intercellular communication (cells no longer communicate effectively, leading to inefficiencies)
• chronic inflammation (constant, low-grade immune response)
• dysbiosis (imbalance of microbes)

This blog post will focus primarily on stem cell exhaustion.

What is stem cell exhaustion and how does it accelerate aging?

As you age, your body’s repair cells, known as stem cells, slowly lose their ability to regenerate tissue. Before they are “exhausted,” stem cells replace worn-down cells, repair damage, and respond to injury. They can make more of themselves (self-renewal) and turn into different types of specialized cells (differentiation). However, over time, they decrease in number, divide less effectively, lose functionality, and even accumulate DNA damage. They become less efficient at repairing your tissues. This is why you experience thinning hair, slower wound healing, muscle loss, and a weaker immune system as you age. Your stem cells have become exhausted. Other hallmarks of aging contribute to stem cell exhaustion, which in turn accentuates other hallmarks of aging, creating a perpetuating cycle of decline.

Examples of stem cell exhaustion and their effects

• Hematopoietic (blood) stem cells (HSCs)

The decline in HSCs weakens the immune system, increasing infection risk and reducing vaccine effectiveness.

• Satellite cells (muscle)

With decreased repair of muscle, there is reduced strength and slower recovery.

• Skin cells

Skin can take longer to heal from cuts and bruises, as well as lose elasticity and wrinkle.

• Intestinal stem cells (gut)

If the gut cannot regenerate, nutrients are absorbed less efficiently, and the permeability of the intestinal lining is increased. This leads to inflammation and dysbiosis – two other hallmarks of aging.

• Neural stem cells (brain)

With reduced neurogenesis (new neuron formation), memory declines.

• Hair follicles

Hair can stop growing and become thin.

• Mesenchymal stem cells (bone)

Bones are constantly adapting to stress and being remodelled. If bone formation cannot keep up with bone breakdown, the risk of osteoporosis and stress fractures increase.

Slowing stem cell exhaustion

It is hypothesized that if a hallmark of aging can be ameliorated (i.e. if stem cell function can be restored), the aging process can be slowed, stopped, or even reversed. The discovery of such an anti-aging drug would be extremely profitable, and this has created the conditions for profit-driven research models, rushed experiments and patenting, false claims and overhype. It is unlikely there will be a one-drug solution to biological aging. Although some therapeutic interventions have shown promise in animals, they are still experimental in humans.

The most studied longevity drugs target pathways, such as mTOR and AMPK. mTOR inhibitors, such as rapamycin, may stimulate autophagy and maintain stem cell function. Signs of aging accumulate as cells divide and grow, and mTOR is a growth signal, so the theory is that inhibiting it will prioritize maintenance and repair overgrowth and reproduction, thereby slowing aging. Although rapamycin has been studied in small clinical trials and is regularly used off label, there haven’t been any large, long-term randomized controlled trials confirming its anti-aging effects in humans. Unlike mTOR, AMPK signals repair and recycling, so compounds that activate it, such as metformin or Berberine, interest researchers and biotech companies. Metformin has been widely studied for type 2 diabetes, but its aging-related outcomes are still developing.

NAD+ boosters, sold as capsules, powders, IVs, injections, drinks, etc., are trending in the mainstream wellness world, hitting US$3.4B sales globally in 2024. NAD+ is an essential coenzyme that helps make energy and repair DNA. It declines as we age, but consuming or injecting it doesn’t necessarily increase lifespan.

There is also a class of drugs known as senolytics. Senolytics kill senescent cells, which are “zombie cells” that no longer divide but release inflammatory signals. It is thought that this will slow aging, and animal trials show compounds like quercetin and fisetin may improve stem cell function. Despite the buzz around senolytics, further research on humans is needed.

Yamanaka factors are genes that can reset a mature cell back into an embryonic-like state, creating induced pluripotent stem cells (iPSCs). The man who identified these genes, Dr. Shinya Yamanaka, won a Nobel Prize for his discovery because it showed mature cells can be fully reprogrammed, which might not just slow aging, but could reverse it. iPSCs can regenerate tissue but, unfortunately, Yamanaka factors aren’t without risks, such as uncontrolled cell growth and cancer.

Many lifestyle interventions have gained traction in the wellness world, such as caloric restriction and exercise. One of the most validated ways to increase longevity is caloric restriction. Eating less inhibits mTOR, activates AMPK, and increases autophagy. But, over time, it can also lower muscle mass and disrupt hormones. Exercise – arguably one of the most powerful aging interventions for humans – has been shown to lower the risk of death. It reduces inflammation, supports stem cell function, promotes neurogenesis, and improves brain health. Unfortunately for investors, lifestyle changes like exercise have the most evidence in humans but aren’t patentable.

Today, billions of dollars are invested in longevity research. However, commercial incentives can lead to misinformation and the overstatement of findings. It is important to be cautious when it comes to products that claim to increase lifespan or reverse aging. Exciting research in the past three decades has shown that it is possible to do so, but human trials are still being conducted.

A scientist analyzing microscopy data. Photo by Faustina Okeke on Unsplash.

Cell therapy manufacturing generates large volumes of process data – from sensors, bioreactors, quality controls and genomic assays. In many facilities, much of that data are collected for regulatory compliance rather than for process improvement – a pattern that continues even as data volumes and sources grow.

The gap between data collected and data used has been identified as a recurring challenge in the field. Addressing it through better analytics tools, clearer data practices and workforce training may help improve manufacturing consistency and, in turn, patient access to therapies.

The data gap in biomanufacturing

The U.S. Food and Drug Administration (FDA) has framed data use as central to its Quality Management Maturity (QMM) initiative, which describes a progression from reactive, compliance-driven data practices toward a state where data actively inform manufacturing decisions. The framework identifies data literacy – the ability of manufacturing teams to interpret and act on process data – as a key factor in that progression.

Manufacturing performance and patient access

Autologous batch failure rates have been reported at up to 25 per cent for some indications, with each failure representing both a financial loss and a patient who does not receive their planned therapy. Research published in JCO Clinical Cancer Informatics found that increasing CAR T wait times from one to nine months raised predicted one-year mortality from 36 per cent to 76 per cent.

Data presented at the American Society of Hematology annual meeting found that approximately 26 per cent of myeloma patients died while waiting for commercially available CAR-T therapy, with limited manufacturing capacity and slot availability cited as contributing factors. These findings illustrate how manufacturing timelines can directly affect patient outcomes.

How data analytics are being applied

Automation and real-time monitoring are being explored as ways to reduce the manual handling steps associated with process variability. Process analytical technology (PAT) tools and machine learning models for metabolic pathway optimization are also moving from academic settings onto commercial manufacturing floors.

Several CDMOs and bioprocessing organizations in Canada and internationally are piloting these tools in cell therapy and biologics manufacturing, and full integration of these approaches across production workflows remains a work in progress industry-wide.

Canada’s position in the field

Canada has built relevant infrastructure and institutional capacity in cell and gene therapy manufacturing, though it operates in a global market where research and manufacturing investments vary considerably by jurisdiction. The Next Generation Manufacturing Canada (NGen) 2025–2026 Corporate Plan notes that scaling advanced manufacturing in Canada will require continued public and private investment to remain competitive internationally.

In 2023, Toronto, Ontario, was home to approximately 1,400 life-science businesses employing over 30,000 professionals. The Pan-Canadian Artificial Intelligence Strategy has directed investment toward AI adoption across key sectors, and NGen has set targets of CA$1.3 billion in total innovation investments and 15,000 new direct jobs by 2028. Programs like CanPRIME, CATTI and CASTL are working to address the workforce skills gap in biomanufacturing.

Organizations such as CCRM, NGen and BioCanRx are working to connect research, manufacturing and commercialization within the Canadian ecosystem. Whether those efforts translate into durable competitive strength will depend on continued investment and regulatory clarity.

The patient dimension

The figures on batch failures and waitlist mortality illustrate a direct connection between manufacturing performance and patient outcomes. When failures rise, patients lose their treatment slot. When release timelines extend, patients in serious condition wait longer.

Researchers have noted that improving manufacturing reproducibility and reducing process variability – as discussed in the Cell & Gene article cited above – are among the factors most directly linked to expanding patient access to cell and gene therapies. Better data practices are one component of that, alongside investment in capacity, workforce and regulatory processes.

Looking ahead

The AI-powered cell and gene therapy manufacturing market is projected to grow from approximately US$14.69 billion in 2025 to over US$122 billion by 2034. Which organizations and jurisdictions develop the manufacturing infrastructure and workforce capability to serve that demand is an open question.

Canada has research institutions, manufacturing capacity under development and many organizations working to connect those assets. Converting that into consistent industrial capability will require coordinated investment over time.

The tools for better data use in biomanufacturing are available and are being tested in practice. The evidence base for their value – as seen in the Blood Advances study cited above – continues to grow. How widely they are adopted will depend on investment decisions, workforce development and the degree to which data capabilities are built into manufacturing operations from the outset.

Professor Samuel Stupp, Northwestern University

Spontaneous neuronal regrowth and repair are not observed in adult spinal cords, making paralysis from spinal cord injury devastating and often permanent.

In a new study, Professor Samuel Stupp’s research team at Northwestern University created human spinal cord organoids (miniature organs derived from human induced pluripotent stem cells) to model different types of spinal cord injuries and test a promising new regenerative therapy.

While many researchers have developed human organoids to study the physiology and pathology of the spinal cord, Stupp’s group is using them to explore a more ambitious possibility: repairing paralyzing injuries in humans.

In an article published recently in Nature Biomedical Engineering, lead author Dr. Nozomu Takata and his colleagues demonstrated for the first time that human spinal cord organoids can reproduce key features of spinal cord injury in humans, including cell death, inflammation, and glial scarring (the creation of a dense mass of glial cells that acts as a physical and chemical barrier to nerve regeneration). Their treatment suppressed glial scar formation, reduced inflammation associated with injury and promoted significant axonal regeneration.

Dr. Nozomu Takata, Northwestern University

I interviewed Dr. Takata on the therapeutic which the lab calls “dancing molecules.” Dr. Takata told me he began his work in Japan, where he was among the first researchers to use principles of developmental biology to re-create organ formation in a Petri dish. After continuing his research in Chicago, at the Center for Regenerative Nanomedicine, he realized that the next logical step, after years of studying human organoids, was to create organoid models of traumatic injuries by damaging them and using them to validate a regenerative therapy.

Dr. Takata modelled injury in two ways: a simple laceration to test the resulting damage; and a contusion. The contusion model closely mimics the injuries people often sustain in car accidents or severe falls. Because the timing and force of the compression can be precisely controlled, the injury is highly reproducible in the lab. Both injury types produced hallmark effects of spinal cord trauma – cell death and the formation of a glial scar.

After establishing a mature spinal cord organoid, Dr. Takata wanted to examine the effect of subsequent spinal cord treatment using the lab’s platform of supramolecular therapeutic peptides called “dancing molecules.”

Fluorescent micrographs. Left: human spinal cord organoid treated with fast-moving “dancing molecules,” showing increased neurite outgrowth. Right: human spinal cord organoid treated with slow-moving “dancing molecules.” Neurite outgrowth is much more subdued than the organoid on the left. Images: Stupp Lab

What are dancing molecules? 

First introduced in a 2021 Science paper by the Stupp lab, a single injection of dancing molecules therapy had been shown to harness molecular motion to trigger axonal growth, prevent glial scarring, and promote functional recovery in mice after traumatic spinal cord injuries (meaning they had recovered some of their locomotion after injury).

The term “dancing molecules” refers to the dynamic motion of molecules within a nanostructure. Although the molecules assemble into stable fibres, the hydrogen bonds and other non-covalent interactions that hold them together are relatively weak and transient. This allows the molecules to move while maintaining their overall nanostructure, enabling them to interact more effectively with receptors on the surface of cells.

In the interview, I also spoke to Dr. Liam Palmer, Research Professor of Chemistry and Director of Research at the Center for Regenerative Nanomedicine. “The molecules may pop up a little or shift slightly,” explained Dr. Palmer, “allowing them to reorganize, or ‘dance,’ to optimize their geometry for binding to cell surface receptors.” Dr. Palmer is a co-author of the paper.

The researchers also examined how the speed of this molecular motion affects signalling. By adjusting the strength of hydrogen bonding, they could fine-tune how freely the molecules moved. When the bonds were weaker, the molecules moved more rapidly, increasing opportunities to interact with receptors and improving cell signalling.

“By dialling back the hydrogen bonding, it allows the molecules to move around more, giving more opportunities for interaction with receptors. The cell signalling is better when the molecular motion is designed to be fast rather than slow,” said Dr. Palmer.

Once these supramolecular peptides engage cell receptors, they trigger regenerative processes such as reduced scar formation, reconnection of axons, repair of neural networks, and improvement of locomotion in vivo in preclinical animal models of spinal cord injury. The tissue regeneration after spinal cord damage was also seen in their human spinal cord organoid.

Producing a regenerative environment

One of the critical receptors that the dancing molecules bind to is called beta-1 integrin – it is one of many ways neurons interact with their environment. Once the molecules bind to this receptor, a cascade of signals that promotes regeneration begins.

Developmental biology inspired this insight into what would allow for a regenerative environment for the spinal cord. Prenatally, when growth of the spinal cord is rampant, there is higher expression of beta-1 integrin present on the neurons, whereas in adulthood, integrin expression declines significantly. This decline provides a clue as to why the adult spinal cord has limited to no ability to repair itself after injury.

“But if we use very bioactive integrin stimulation using our nanomaterials reported in the Science paper [in] 2021,” Dr. Takata said, “we may be able to help adult neurons grow again.”

Dr. Palmer describes the strategy more simply: “We’re essentially tricking the nervous system into thinking it’s in a regenerative environment.”

The first spinal cord injury model with an immune component 

This research is also the first to incorporate microglia – a type of immune cell – into a central nervous system organoid injury model, simulating the inflammatory response seen in human traumatic spinal cord injury.

Dr. Takata explained that the contusion model of spinal cord injury involves mechanically compressing the human organoid tissue to create traumatic damage. “That causes cells to die, just like what would happen in a person who suffered a traumatic spinal cord injury. The dying cells is [sic] a message for the immune cells to be activated,” Dr. Takata said. Once activated, the microglia release factors that trigger astrocytes to form a glial scar, which is the dense tissue around the injury site. The astrocytes also secrete chondroitin sulfate proteoglycan, a chemical that inhibits neural regeneration.

By adding microglia, the organoid becomes a more biologically realistic platform for testing translational therapies, particularly because immune responses are one of the major pathological drivers of scar formation as barriers to regeneration after human spinal cord injury.

Clinical trials

For a person with a spinal cord injury, the researchers hope that a single injection of their dancing molecule therapy will allow for regeneration.

Within 24 hours of a severe spinal cord injury, neurosurgeons commonly perform decompression surgery to remove bone fragments and relieve pressure on the spinal cord. This procedure could also provide an ideal opportunity to administer the therapy directly to the injury site, potentially preventing the formation of the initial glial scar.

Human organoids provide one of the closest experimental models to the human spinal cord, making them an important step toward clinical translation. The researchers are currently working with the U.S. Food and Drug Administration to advance the therapy toward clinical trials. The agency has already granted the treatment Orphan Drug Designation.

“We think it’s still at least a year away,” Dr. Palmer said, “but we’re getting closer to a human trial. There are still many safety and efficacy studies underway, and we’re working through them now.”

Infographic provided by the author

Science communication sits at the intersection of research, society and decision-making. However, in recent years, the space has become more crowded with artificial intelligence (AI)-generated content, influencer commentaries and monetized narratives, which are reshaping how science is interpreted, trusted and used. Both the creation and consumption of science communication are fundamentally changing.

In 2026, six trends are shaping how science, including advances in regenerative medicine and stem cell research, is explained and experienced.

Whether you are a science communicator yourself, or a regular consumer of news and content, this post will provide you with a roadmap for where the science communication field is going.

Trend 1: AI-driven discovery

AI is transforming how audiences find and engage with science. Science communicators are using AI tools to generate visuals, summaries and narratives from complex research, ensuring they resonate with the target audience(s). Platforms like Reelmind.ai can automatically convert datasets into animated visuals, explainer videos and interactive models, making dense research easier to understand and share. Other tools like ChartGPT and Google Charts help turn raw data into charts and narratives that resonate without advanced coding.

While AI can help to simplify, summarize and tailor information and content, communicators must also be aware of the potential harms to society that it presents. For example, using a third party to verify facts that AI presents can reduce the risk of publishing AI’s “hallucinations.” Read more about the challenges and opportunities of AI in science communication from the Stanford Social Innovation Review.

Trend 2: Visual and immersive storytelling

Visual formats are essential for breaking down complexity. Communicators are leveraging infographics and interactive graphics to make data compelling and accessible. For example, the Knight Lab at Northwestern University provides free tools, including Timeline for interactive timelines, and Juxtapose, for before-and-after comparisons, which are popular ways to illustrate change and process visually.

Frameworks and tools, like the “Using Multimedia & Visuals” section of the American Association for the Advancement of Science’s Communication Toolkit, help science communicators plan and produce high-impact visualizations.

Trend 3: Trust and transparency

In an October 2025 panel discussion called “Trust Issues: Science, Skepticism & Showing Up,” hosted by RCIScience, a panellist stated a key maxim of science communication: “The idea of transparency is indispensable to building trust.”

Audiences increasingly expect clarity about uncertainty, methodology and context. Responsible science communication, supported by organizations such as OECD.AI, emphasizes accurate and transparent explanations of scientific advances, avoiding hype and clearly stating limitations.

Tools that help annotate visuals with citations or versions, such as collaborative commenting systems in visualization platforms, support transparency by allowing audiences to trace back to original data and methods.

Trend 4: Patient- and clinician-relevant context

Effective science communication explains not just what was discovered, but what it means for real-world decisions. Narratives that combine patient experience with clear data interpretation help bridge research and practice.

Visual analytics platforms like TrialView demonstrate how clinical trial data can be presented interactively, helping clinicians and patients explore patterns and outcomes in an intuitive way.

Trend 5: Inclusivity and global perspective

Inclusive communication expands beyond translation to cultural context and accessibility. Tools that adapt content to include features like multilingual captions and sign language support help broaden reach. AI translation and adaptive glossaries in platforms like Reelmind.ai (also mentioned under the first trend above) can also assist these efforts by localizing science in multiple languages and literacy levels.

Inclusive practices also mean designing for diverse learning needs, whether through captioned videos, accessible websites or culturally relevant examples.

Trend 6: Open science and communication

As per the Government of Canada’s website, open science is the “practice of making scientific inputs, outputs and processes freely available to all with minimal restrictions.” It influences how research is shared and used. Science communicators play a crucial role in helping audiences interpret open science outputs, explaining uncertainty and the evolving nature of evidence.

Open science is not new, but I’m highlighting tools to assist science communicators. Common tools include the Open Research Knowledge Graph, which creates structured, machine-readable representations of research contributions, making it easier to compare findings, discover methods, and build narratives around evidence. Also, preprint servers such as bioRxiv and medRxiv accelerate dissemination, while open-access journals like PLOS One break down paywalls for broader public access.

Looking ahead, science communication will be less about broadcasting results and more about helping audiences understand and engage with science responsibly. By understanding and using AI tools, visual storytelling, trust-building practices, relevance, inclusivity and open science principles, communicators can connect complex research to society in meaningful ways.

If you’re aware of a new or emerging trend or tool that I’ve missed, please share it in the comments.

Increasingly, that future doesn’t just look like roads and buildings. It looks like regenerative medicine, advanced manufacturing and biomedical devices that blur the line between engineering and human health. That distinction matters.

The recent past

Over the past two decades, women have made measurable – and meaningful – gains in engineering. Undergraduate enrolment has climbed steadily, reaching just over 25 per cent nationally in 2023. In some disciplines – particularly biosystems, environmental and chemical engineering – women now represent a significant share, in some cases approaching or exceeding 40-50 per cent.

Notably, many of these gains are concentrated in fields that sit at the intersection of engineering and life sciences. Biomedical engineering, regenerative medicine, and biofabrication have emerged as areas where women are not only participating, but shaping the direction of research and innovation. From tissue engineering and 3D bioprinting to implantable devices and precision drug delivery, these fields are redefining what engineering looks like and who sees themselves in it.

In other words, the pipeline is no longer the problem it once was. But beyond graduation, the story becomes more complicated.

Despite increased participation at the entry level, women still make up only about 15 per cent of engineering professionals in Canada, according to a report from the Diversity Institute at Toronto Metropolitan University (TMU). The drop-off is real and persistent. In Ontario, for example, women represent roughly one in five engineering graduates, but are less likely than men to end up working in engineering roles at all, according to a report by Wendy Cukier for the Council of Canadian Academies (CCA) on the “State of equity, diversity, and inclusion within Canada’s science, technology and innovation ecosystem.”

This is the “leaky pipeline” we keep talking about. And it leaks at multiple points. There are the familiar factors: workplace culture, bias in hiring and promotion, and the disproportionate burden of unpaid care work. Many women still describe engineering environments as legacy systems that are slow to change, and often shaped by long-standing networks that are difficult to access from the outside, as per the TMU report above.

Not surprisingly, compensation is still an issue. By 2021, the gender pay gap for engineering graduates in Canada had widened significantly, with women earning roughly 75 per cent of what their male counterparts earn on average, says the CCA. That gap compounds over time, influencing everything from career mobility to leadership opportunities.

And leadership is where the gap becomes most visible.

Again from TMU, women hold only about 18 per cent of engineering management roles. In academia, the numbers are similar or worse at senior levels. Even in research-heavy fields like biomedical engineering, where participation is higher, women are less likely to occupy the most senior, decision-making positions or to be recognized as lead authors on major publications. That means representation is improving at the front end, but influence remains uneven.

The present and the future

Engineering is in the middle of a transformation. For example, advanced manufacturing is becoming more automated, data-driven and digitally integrated. Biomedical engineering is converging with artificial intelligence (AI) to enable everything from predictive diagnostics to personalized treatment design.

Jobs are changing and will continue to do so, but AI opens up opportunities for new ones, some that are obvious and some that we haven’t even imagined yet. When the Internet began reshaping the field of public relations, social media managers didn’t yet exist. The same will happen as a result of AI; fortunately, humans are resilient and we will adapt. Engineering jobs are not disappearing so much as evolving, especially at the entry level.

If fewer traditional entry-level roles exist, access to meaningful early-career experience becomes more competitive, which could amplify existing inequities. Those with stronger networks, better mentorship, or fewer systemic barriers will have an advantage. Without intentional intervention, the same gaps we see today in jobs, salaries, and leadership could widen.

On the bright side, fields like regenerative medicine and biomedical engineering are still being somewhat defined. Advanced manufacturing is being rebuilt around digital tools and new workflows. These are not legacy systems in the same way as traditional engineering disciplines and that offers a real opportunity to build them differently.

Ideally, we will see more inclusive hiring practices and transparent compensation structures – something that the Government of Ontario now requires. There’s also an opportunity to develop more accessible leadership pathways and mentorship can really help those at the early-career stage.

Getting women into engineering matters, but ensuring they stay, advance and lead is equally important.

Today’s engineering jobs are complex and solving them will require the full spectrum of talent (i.e. representation).

National Engineering Month is a moment to celebrate progress, but it’s also a moment to be honest about what remains unfinished. The question is not just what we are building but who gets to build it. As a mother of a daughter who is about to graduate with an engineering degree, that matters a lot.

Are you ready to celebrate engineers? Watch this stand-up comedy routine by engineer Don McMillan.

A CCRM scientist looking through a microscope

Somehow we are already into March, and spring – at least in some places – feels close by. To catch you up, I’ve put together a recap of some of the research that scientists were buzzing about in the field of regenerative medicine from 2025; I cover the good, the bad, and the controversial. All of these stories were first highlighted in Regenerative Medicine News Under the Microscope, so consider this a greatest-hits compilation. These investigations just continue to get bigger, and it’s very encouraging to think that we’re finally making some progress in bringing what could be game-changing treatments out of labs and to people who really need them.

Let’s jump right in! In no particular order:

1. Parkinson’s disease

Specifically, work by Bluerock Therapeutics, which announced that their stem cell therapy for Parkinson’s would be moving to Phase III. It was found to be safe, and there are hints of potential efficacy, but we now get to find out how effective it really is with more patients trying out the treatment.

2. Good news for epilepsy

Another stem cell therapy that jumped to Phase III this past year was Neurona Therapeutics’ solution for epileptic seizures that don’t really respond to medications. This one’s particularly interesting because we finally get to test theories that have been investigated in vitro and in animals for years. In its simplest form, the theory is that epilepsy is likely caused by an imbalance between the chemicals in the brain that amplify or increase electrical activity, and the chemicals that dial down or inhibit activity. It continues that epilepsy patients may not have enough of the dampening chemical, and there’s just too much runaway electricity. Neurona’s cell therapy introduces more cells into the brain that produce the inhibitory chemical, just to dial things down a bit, and again it was looking very promising in the Phase I/II trial.

3. Vision repair

Optogenetics is one of my specialties, so stories like these are always particularly exciting to me. In 2025, there was a very elegant application of optogenetics for vision loss. Patients received a genetic therapy involving a lab-created, light-sensitive protein. I would so encourage our readers to go and watch the videos included in the published paper; it was really inspiring to see someone go from not being able to detect where a light is in a room to actually being able to walk straight towards it.

4. Healing damaged corneas with one’s own stem cells

This story is really USA-specific. Researchers conducted a Phase I/II clinical trial of the first xenobiotic-free, serum-free, antibiotic-free manufacturing protocol developed in the U.S.

While this paper is exciting, I just wanted to highlight that using healthy limbal stem cells from one eye to heal a damaged cornea in the other is not new, and the authors don’t claim it to be so, yet the news on this story was largely making it out to be an entirely novel procedure; I thought this was interesting. In fact, we’ve been doing it here in Canada for a while. The actual novelty offered by this protocol is that manufacturing guidelines in the U.S. are different, and that’s what’s preventing autologous limbal stem cell transplants in their current form from being used by our Southern neighbours… until now, potentially. The researchers behind this paper modified various in vitro steps to adhere to the U.S. Food and Drug Administration’s (FDA) Good Manufacturing Practices, meaning this treatment might soon be accessible to patients in the U.S. – a great accomplishment.

5. Stem cells for diabetes

Vertex Therapeutics, which also manufactures Casgevy – the gene-editing medicine for sickle cell disease – has also developed a cell therapy for severe type 1 diabetes, and there are people who have participated in their clinical trial who are now able to make their own insulin. The data was published in 2025.

Significant downside: Patients receiving this therapy also need to take immunosuppressants because the cell therapy comes from someone else. To get around this, Vertex was also working on a different version of this treatment where the transplanted cells could be encapsulated in a device that would protect them from the immune system. Unfortunately, it was announced last year that their device failed in a separate clinical trial so, for now, immunosuppression is the only option.

6. Alzheimer’s disease

Longeveron published its Phase IIa CLEAR MIND trial with positive results, testing a mesenchymal stem cell (MSC) therapy for mild Alzheimer’s disease (AD). This was an intravenous treatment created using bone marrow-derived MSCs.

MSCs are being tested for many different diseases because they seem to be really adept at modulating the immune system and managing inflammation (and as you likely know, what disease these days doesn’t involve inflammation). Their goal was to slow the clinical progression of AD and, importantly, immunosuppressive drugs weren’t required in this trial despite the fact that the cells did come from donors. This is because MSCs are immunoprivileged, and they don’t trigger the immune system in the same way other cells might.

A Phase II/III trial is currently in development.

7. A darker story about Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a genetic disorder that causes muscle degeneration. You can imagine how that affects everything in the body: one’s ability to move, breathe, keep the heart beating properly, all of it. A company called Sarepta Therapeutics created a one-time genetic therapy for the disease, and a few deaths in 2025 revealed that it’s really not for everyone with DMD. Patients who were walking around on their own were able to take this medicine largely without major incidents. However, a pair of patients did die after taking this medicine, and it turns out they were non-ambulatory, meaning they couldn’t walk independently. The FDA took this information and decided to restrict use of this medication to only those who could walk, and walking here appears to be a readout of disease severity. Liver failure was the cause of death, a complication of viral gene delivery. The FDA also added a warning to the label for patients with pre-existing liver issues.

Fierce Pharma has a great piece on this story if you want to learn more.

8. Onto more positive news: custom CRISPR medicines

Six months, one week, and four days.

That’s how long it took scientists to create a custom CRISPR therapy for baby KJ, who was suffering from a rare, life-threatening condition called CPS1 deficiency – short for carbamoyl phosphate synthetase I deficiency. This timeline is unprecedented, given that drug development usually takes over 10 years.

CPS1 deficiency is a genetic defect of the process by which the body metabolizes protein. What results is a buildup of ammonia in the tissue, which can be toxic (especially in the brain). Estimated to have an infant mortality rate of 50 per cent and affect just 1 in 1,300,000 people, even if the child does survive, there’s still a risk of developmental delays and intellectual disabilities.

Of course, once the baby is diagnosed, their protein consumption is often significantly restricted and nitrogen scavenging medication is usually prescribed. In KJ’s case, rather than the usual liver transplant that would follow, the research team of Musunuru et al. developed a custom CRISPR therapeutic to edit the infant’s genetic code.

To date, KJ has been stable and is consuming more protein and less medication, suggesting efficacy. However, longer follow-up will be required to assess the overall safety and determine just how well the treatment works. So far, so good!

Read more in this NIH news release. 

9. CAR T-cell therapy

As you likely know, CAR T-cell therapies available in clinics are currently ex vivo: T cells are removed from the body, upgraded in the lab, then returned to the patient.

Taking this strategy to the next level, researchers have been working on a way to upgrade T cells without removing them first. A very nice example of this was published by Hunter et al. Their team developed an in vivo genetic engineering strategy to reset the immune system and create CAR T cells in humanized mice and cynomolgus monkeys.

They use targeted lipid nanoparticles to deliver the mRNA to specific subsets of T cells.

Check out this great news piece in Nature Reviews Drug Discovery that summarizes progress in this space.

10. Cancer vaccines!

This piece wouldn’t have been complete without mention of Anixa Biosciences’ breast cancer vaccine.

Phase I enrollment wrapped up in 2025. The vaccine is a set of three shots given two weeks apart. So far, 35 women have received the vaccine.

Preliminary results suggest that the vaccine is well-tolerated and that it does indeed mobilize an immune response, so Phase II is in the works.

11. Aging

A single factor for safer cellular rejuvenation.

This paper left everyone in the scientific community very confused. A company called Shift Bioscience found a gene that they claimed lowers single-cell age and attenuates harmful signatures of multiple hallmarks of aging.

They proceed not to tell us what that gene is.

It’s really not the norm to publish a “scientific” paper that keeps the gene being studied entirely hidden. I understand that the team is treating it as a trade secret for business reasons, but asking for blind trust at any stage is unreasonable, especially in the wake of other controversies in the regenerative medicine field. I doubt the paper will get accepted into a mainstream journal as-is for this reason; it’s currently a pre-print.

It’s especially strange as a strategy because this is what patents are for. If the team can produce an intervention to manipulate the gene, they can file a patent like any other biotech company. It seems like they’re intentionally trying to spur speculation and suspense, so… moving on.

12. We’re ending on a high note with Down syndrome

CRISPR was used in vitro on human cells to correct the chromosomal abnormality (a third copy of the 21^st chromosome) that causes Down syndrome and restore cell function. This is very promising work that serves as a proof-of-concept for future therapeutic avenues.

 I hope this recap was helpful and that the year is off to a great start for you all!

Live microbes on a Petri dish prior to genetic engineering. Image: Pixabay

Microbiota-based products, supplements, or therapies can be used in combination with stem cell treatments to create a more hospitable and welcoming environment for transplanted stem cells to take hold and grow. When used in conjunction with stem cell treatments, these microbiota-based therapies are known as microbial adjuncts, non-essential but beneficial additions to the primary treatment (in this case, stem cell transplantation).

Microbial adjuncts can calm the immune system, thereby preventing unnecessary attacks on transplanted stem cells. They can be broken down into five categories: metabolites/postbiotics, live microbes/probiotics, microbiome conditioning, ex vivo stem cell priming, and bioengineered microbes. All five categories of microbial adjuncts enhance stem cell therapies by modulating the immune system, promoting stem cell proliferation and differentiation, improving engraftment, and supporting healthy tissue via signalling molecules and metabolites.

Metabolites/postbiotics

When microbes undergo metabolism, they produce what are known as “metabolites.” These are also referred to as postbiotics. They are the non-living signalling molecules and/or chemical messengers of live bacteria. Some of the most abundant microbial metabolites are short-chain fatty acids (SCFAs), such as butyrate, acetate and propionate. Gut microbes produce SCFAs when they ferment fibre. SCFAs reduce inflammation and calm the immune system by promoting regulatory T cells; this allows stem cells to grow in a healthy environment. They also influence which specific cell type stem cells will become once they differentiate.

Other metabolites include tryptophan derivatives, B vitamins and lipid mediators, amongst many others. These all play a role in immune modulation and regulation, and each has a wide range of subtle effects on the host. Because they are stable, controllable, non-living, and targetable to specific functions, metabolites are the safest type of microbial adjunct.

Live microbes/probiotics

Patients undergoing stem cell therapies can receive live microbes, rather than the metabolites of live microbes. They are generally considered less safe than metabolites due to their unstable and living nature. In immunocompromised patients, there is a risk of infection when live microbes are administered. Additionally, different species and strains have different effects on host physiology, depending on a variety of factors, some not yet elucidated.

It is unclear whether probiotic supplementation is transient or if the microbes are able to assimilate into the microbiome, altering the composition chronically rather than acutely. It might be species-specific, like if some can survive more than others through stomach acid, eventually making their way to the colon. Usually, strains of Lactobacillus and Bifidobacterium are used, as these have shown promise in clinical studies to promote stem cell proliferation and differentiation, support gut health, and reduce the risk of graft-versus-host disease (GVHD).

Microbiome conditioning

Microbiome conditioning encapsulates a variety of therapies that modulate the microbiome, ranging from diet interventions to fecal microbiota transplants. For example, increasing fibre in the diet can lead to an increased production of SCFAs, which reduce inflammation, as mentioned above. Additionally, fibre intake can be increased via prebiotic supplementation; prebiotics are non-digestible fibres that feed beneficial bacteria in the gut. An increase in these beneficial bacteria also leads to increased SCFA production. Similarly, increasing antioxidants in the diet can foster an ideal environment for stem cell proliferation and differentiation by reducing oxidative stress and supporting the metabolic functioning of the host.

Fecal microbiota transplants (FMTs) are a higher-risk intervention than diet changes. FMTs involve taking the entire gut microbiome composition of a healthy individual (via fecal matter) and transplanting the fecal matter into the patient’s colon. There is not yet a standardized procedure for FMTs, and there remain risks for infection, particularly in immunocompromised patients. However, when successful, gut health can be restored, and the risk of GVHD can be greatly reduced or even eradicated.

Microbiome conditioning interventions are personalized and patient specific. With something as complex as the microbiome, some interventions could unintentionally be doing more harm than good. More research is needed, but it is difficult to perform when microbial communities in the human gut are constantly evolving and communicating in myriad ways, with both each other and their human host.

Ex vivo stem cell priming

Ex vivo stem cell priming is treating stem cells outside the body before they are transplanted. The goal is to make them more effective once they are infused into the body. One way to do so is with microbial metabolites. For example, hematopoietic stem cells can be modulated with SCFAs to influence differentiation, which is what stem cells decide to become once transplanted. Although research for microbial priming of stem cells is still preclinical at this stage, and limited to mice, it is an exciting and trendy area.

Bioengineered microbes

The bioengineering of live microbes is currently one of the most cutting-edge niches of synthetic biology, an area of science focused on designing biological parts or entities. Some ambitious and ongoing goals of synthetic biology are creating bacteria that can digest plastic, or engineering organisms that can produce fuel. The regulation of synthetic biology is messy, however, due to the fact that bioengineering leads to genetically modified organisms (GMOs). Therefore, bioengineered microbes are far from clinical use and are likely a therapy of the future. Nonetheless, they could offer a more targeted approach than typical probiotic supplementation, as long as off-target effects are mitigated. Bacteria could be modified to produce specific metabolites that benefit stem cell treatments (i.e. bacteria that produce VEGF and thereby increase angiogenesis of mesenchymal stromal cells).

In conclusion, the microbiome can be harnessed to improve the efficacy of stem cell therapies, largely by promoting proper immune tolerance, which in turn reduces inflammation, lowers the risk of GVHD, and supports healthy tissue growth and repair. But harnessing the microbiome isn’t easy, with the exasperating complexity of microbe-microbe interactions and microbe-host interactions, usually via metabolites. It is essential that we do not underestimate or overestimate the power of synthetic biology and bioengineering of complicated microbiota compositions, as bacteria have been around for billions of years and are capable of horizontal gene transfer and rapid evolution. The immune system and the microbes that live in and on us are so intricately connected, which is what makes microbiome modulation and microbial adjuncts such a strong determinant of immune function, inflammation, and stem cell transplantation success.

Image created through ChatGPT

In 2025, cell and gene therapy (CGT) showed growing clinical familiarity even as access and reimbursement challenges persisted. Providers and payers increasingly trusted and valued CGTs, supporting the continued expansion of late-stage programs. However, startup activity remained muted amid economic uncertainty, regulatory shifts, evolving therapeutic priorities and increasingly selective investors. Even though total deal value increased, the number of deals remained low, as buyers focused on fewer transactions that closely aligned with their priorities and relied more on milestone-based payments. The EY Firepower report described 2025 as a year of “smaller, smarter” dealmaking, reflecting a more disciplined and intentional approach across the market.

What mattered most was not deal volume but where capital flowed, as investment concentrated around late-stage clinical data, regulatory inflection points and strategic partnerships that signalled de-risked science and commercial readiness.

CGT dealmaking overview by quarter in 2025

In the first quarter (Q1) of 2025, a total of 90 CGT deals were recorded, 20 per cent fewer than in the previous quarter. Despite the overall slowdown, acquisition activity rose from seven to nine deals, reflecting sustained interest in de-risked assets. From a pipeline perspective, 27 non-genetically modified cell therapy trials were initiated in Q1 with 74 per cent targeting non-oncology indications, reflecting growing interest in autoimmune and inflammatory diseases. In contrast, 79 gene therapy trials were launched, with 57 per cent focused on oncology – the highest concentration observed over the past year.

In the second quarter (Q2), overall deal volume stabilized while acquisition activity increased to 12 transactions. This trend highlighted buyers’ growing confidence in deploying capital selectively, even as funding for early-stage companies remained limited. From a pipeline perspective, Q2 showed a renewed emphasis on RNA therapeutics targeting oncology, with the highest proportion of RNA oncology trials recorded in the past two years. This shift reflected increasing confidence in RNA-based approaches beyond vaccines and infectious disease, supported by continued advances in RNA delivery technologies and a clearer line of sight to oncology-focused clinical and commercial value.

By the third quarter (Q3), deal activity rebounded to 99 transactions, driven largely by partnerships and structured deals, whereas acquisitions dropped to just three. Buyers appeared more comfortable sharing risk than committing to outright acquisitions. Scientific momentum remained strong, with 125 trials launched across gene, cell and RNA modalities, indicating continued investment in clinical development even as capital markets grew more selective.

The fourth quarter (Q4) of 2025 showed more active CGT dealmaking, with heavier use of contingent value rights and milestone-based economics as a key signal. According to GlobalData, the average milestone payment for biopharma deals increased in Q4 compared with Q3. At the same time, market sentiment improved as the XBI, a biotech stock index, rose by almost 75 per cent from earlier lows to reach its highest level since 2021.

Which therapeutic areas had the biggest deals in CGT?

Autoimmune

In vivo cell therapy clearly stood out for investors in 2025, as it removes ex vivo manufacturing bottlenecks and makes cell therapy easier to scale. AbbVie acquired Capstan Therapeutics for up to US$2.1 billion to gain its in vivo CAR T platform that engineers immune cells directly inside the body for the treatment of autoimmune diseases and cancer. AstraZeneca followed a similar approach with its US$1 billion acquisition of EsoBiotec, whose ESO-T01 platform reprograms immune cells directly in vivo.

Cardiometabolic

Expanded gene-editing deal activity in cardiometabolic disease reflects a shift toward durable, one-time in vivo genomic therapies to replace lifelong therapies. Eli Lilly’s up to US$1.3 billion acquisition of Verve Therapeutics is centred on VERVE-102, a base-editing therapy designed to permanently lower cholesterol. The deal highlights Lilly’s push to shift cardiovascular care away from lifelong dosing toward durable, single-intervention treatments.

Renal

Addressing the rare genetic disorder autosomal dominant polycystic kidney disease (ADPKD) drove Novartis’ US$1.7 billion acquisition of Regulus Therapeutics. Farabursen is a potential first-in-class oligonucleotide targeting miR-17 with preferential kidney exposure. Phase Ib clinical data showed reductions in cyst growth and kidney volume along with signals suggesting delayed disease progression in ADPKD.

Ophthalmology

Big Pharma made its strongest move into ophthalmology in 2025, where durable, single-dose therapies could replace chronic injection regimens. Eli Lilly agreed to acquire Adverum Biotechnologies in a deal valued at up to US$260 million for Ixo-vec, an intravitreal gene therapy being developed for wet age-related macular degeneration. Just weeks later, Lilly also partnered with MeiraGTx in a deal worth more than US$400 million in potential payments, gaining access to its AAV-AIPL1 program for a severe inherited retinal disease supported by early clinical data.

What to expect in CGT dealmaking in 2026

In 2026, CGT dealmaking is likely to remain highly selective, reflecting the disciplined approach seen in 2025. While innovation will continue to matter, investment decisions will increasingly be shaped by deal structure evolution, international partnerships and shifting therapeutic priorities.

Evolving capital structures

Capital structures in life sciences will continue to evolve as investment capacity aligns more closely with opportunity. Mergers and acquisitions (M&A) activity in 2026 is expected to increase, supported by stabilizing financing conditions and selectively reopening equity markets. As outlined by Amanda Frick, private equity, royalty financing and minority investments are now mainstream tools for bridging late-stage development and commercialization. These structures provide added runway and flexibility for innovators constrained by earnings-per-share considerations, without immediate dilution. This dynamic is expected to persist into 2026, with private equity remaining active across pharma technology.

China-West collaboration

Cross-border collaboration is expected to remain a central driver of CGT dealmaking in 2026, as Chinese biotechs increasingly adopt multi-regional clinical trials and attract early-stage CGT development partnerships abroad. China’s biotech dealmaking reached record levels in 2025, with more than 140 China-related licensing and investment deals announced, and activity is expected to continue rising. Without strategic collaboration, Western markets risk not only losing leadership in innovation but also delaying patient access to emerging CGT therapies.

Therapeutic expansion

CGT will continue moving beyond its early experimental phase. Base editing is expected to play a much larger role as next-generation tools reduce DNA damage and enable multiple genetic modifications simultaneously. At the same time, companion diagnostics will evolve beyond single biomarkers, with multi-omics approaches improving patient selection and accelerating trial execution – making it more realistic to pursue chronic, rare and neurological diseases. Momentum will also continue to build around off-the-shelf immune products and next-generation immunotherapies that can be manufactured and delivered at scale, rather than highly individualized treatments. Developers will face increasing pressure to move away from niche inpatient settings toward cost-constrained outpatient care models, elevating the importance of durability, pricing discipline and health economic justification.

As CGT moves into 2026, the market is shifting from a period of caution to one focused on execution and strategic focus. The message from 2025 was clear: investors backed programs with strong clinical data and a realistic path to market, not scientific promise alone. In 2026, dealmaking is likely to centre on fewer, higher-conviction transactions, supported by more flexible capital structures, stronger cross-border collaboration and a growing focus beyond oncology into more common diseases. Ultimately, the next phase of value creation in CGT will be led by teams that can turn biological breakthroughs into therapies that are scalable, durable and economically viable.