The research team, led by Christopher Walsh, MD, PhD, chief of the Division of Genetics and Genomics at Boston Children’s and an investigator of the Howard Hughes Medical Institute, and collaborators Alice Eunjung Lee, PhD, and August Yue Huang, PhD, also in the Division of Genetics and Genomics—who are all professors at Harvard Medical School and associate members of the Broad Institute of MIT and Harvard—say their study findings may provide insights into new Alzheimer’s disease diagnostics and treatments.

“We find that to some extent, Alzheimer’s disease is a little like cancer—driven by the same mutations that drive blood cancers like lymphoma and leukemia,” said Walsh. “This is helpful because we have a lot of drugs to fight cancer and some of them might be useful therapeutically for Alzheimer’s disease.”

The researchers reported on their work in Cell, in a paper titled “Somatic cancer variants enriched in Alzheimer’s disease microglia-like cells drive inflammatory and proliferative states.”

Microglia function as the brain’s resident immune cells, acting as garbage collectors, eating debris and infected or dying cells. “The importance of microglia in Alzheimer’s disease (AD) pathogenesis has been demonstrated by large-scale genetic association studies, which have identified AD risk variants in a growing list of microglia-related genes,” the authors wrote. “Once abnormally reactive in AD, microglia can promote synaptic and neuronal loss while exacerbating tau proteinopathy.”

Unlike the rest of the immune system cells that circulate in the blood throughout the body, microglia don’t cross the blood brain barrier—or so experts thought. For their newly reported study the research team sequenced 149 cancer-driving genes from tissue samples in 190 brains donated from people with Alzheimer’s disease compared to 121 healthy brains. The Alzheimer’s samples had more single DNA letter changes than the healthy tissue with the most changes found repeatedly in the same five cancer driver genes, meaning the microglia were amassing mutations in specific genes. “Deep (>1,000×) panel sequencing of 311 brain samples revealed enrichment of somatic single-nucleotide variants (sSNVs) in cancer driver genes in AD brains, especially in genes associated with clonal hematopoiesis (CH),” the team stated.

The cancer gene mutations the researchers discovered in the microglia are commonly found in blood cancers. Because of this, the team tested blood samples from people with Alzheimer’s disease for these same mutations. The team didn’t expect the blood to have these mutations. However, Walsh’s team found the blood cells of the same Alzheimer’s patients carried the same cancer mutations too.

The researchers theorize that the blood-brain barrier weakens, either by age or injury, allowing the blood’s immune cells to cross into the brain. These new arrivals then convert into microglia-like cells. Separately, clumps of proteins accumulate in the brain, triggering microglia to proliferate and respond. The cells most likely to dominate are those with a selective advantage, such as the microglia-like cells with the cancer mutations. However, these mutant microglia also make the environment more inflammatory and hostile than that of the healthy microglia, causing innocent bystander neurons to die off, which leads to Alzheimer’s disease. “These findings suggest that clonal somatic driver variants in MLBMs are enriched in AD, potentially promoting neuroinflammation and neurodegeneration,” the researchers noted. “Potential roles of somatic cancer driver variants in AD pathogenesis open up a whole new range of therapeutic avenues in AD, complementary to approaches emphasizing amyloid and tau.”

Lee added, “Because it’s hard to access brain tissue in a living patient, genetic screens using blood samples could be developed to test whether a person carries these mutations, and has an increased risk of developing Alzheimer’s disease.” Lee and Huang performed a follow-up study, now posted as a preprint on bioRxiv. Here, they demonstrated that cancer driver mutations observed in patient blood samples increased risk of Alzheimer’s disease independently of a well-established genetic risk factor, APOE4.

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“Immunotherapy has transformed cancer treatment, but only a subset of patients benefit from it, and predicting who will respond remains challenging,” said Rukhmini Bandyopadhyay, PhD, a postdoctoral fellow at The University of Texas (UT) MD Anderson Cancer Center.  

“This study represents, to our knowledge, the first deep learning-based pathomics biomarker rigorously validated across international real-world cohorts and a Phase III randomized clinical trial, directly addressing one of the most urgent unmet needs in precision oncology: reliable patient selection and stratification for immunotherapy,” he continued. 

Pathomics applies computational and machine learning methods for high-throughput analysis of digital pathology images to extract large-scale data related to cell and tissue architecture linked to disease outcomes. 

Bandyopadhyay and colleagues developed a deep learning survival prediction model called Pathology-driven Immunotherapy Optimization (Path-IO), which can study patterns across tissue to identify patients most likely to benefit from immunotherapy. The model then combines imaging and clinical data to estimate whether a patient may have a higher or lower risk of poor outcomes from immunotherapy. 

The researchers tested the platform in a study that included 797 immune checkpoint inhibitor-treated NSCLC patients from UT MD Anderson, with external validation in 280 additional patients from Mayo Clinic, Gustave Roussy, and the Phase III Lung-MAP S1400I trial in which immunotherapy-naïve patients with lung squamous cell carcinoma, a subtype of NSCLC, were treated with immune checkpoint inhibitors. 

The model reliably stratified patients into higher and lower risk groups. In the UT MD Anderson cohort, patients in the high‑risk group had more than double the risk of death or disease progression compared with patients in the low‑risk group. 

Model performance was evaluated using the concordance index (C-index), which measures how well each biomarker distinguishes between patients with different outcomes. Notably, Path-IO consistently outperformed PD-L1, the U.S. Food and Drug Administration-validated standard-of-care biomarker for guiding immunotherapy use in NSCLC patients, across both discovery and test cohorts.  

PD-L1 alone showed limited prognostic performance, with C-indices of 0.58 for overall survival (OS) and 0.57 for progression-free survival (PFS) in the discovery cohort, declining to 0.50 and 0.51, respectively, in the test cohort. In contrast, Path-IO demonstrated stronger discriminative ability, achieving C-indices of 0.69 for OS and 0.65 for PFS in the discovery cohort and 0.63 for OS and 0.58 for PFS in the test cohort.  

Combining pathology-based predictions with radiomics and clinical data further improved the model’s performance, with the C-index increasing from 0.58 to 0.70 for PFS and from 0.63 to 0.75 for OS.  

Given that the approach was designed to be applied to routine pathology slides, the platform can be incorporated into existing clinical workflows without significant expense compared to other emerging data-based technologies. 

As the study is retrospective, further investigation is needed to go beyond the identification of patients who would benefit from immunotherapy and help predict what type of immunotherapy they can benefit from. Future directions include prospective validation and the integration of paired, more comprehensive molecular profiling to enhance predictive performance.

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Data from Cosmo’s Phase III program for clascoterone confirmed the drug’s long-term safety profile was comparable to vehicle, supporting suitability for chronic use in a lifelong condition. Clascoterone also showed a novel mechanism designed to target the underlying biology of hair loss and continued efficacy with ongoing use, Cosmo said.

A total of 1,465 patients were enrolled in the Phase III program, the largest for any topical treatment candidate for male AGA, according to Cosmo. The program consists of the SCALP 1 (NCT05910450) and SCALP 2 (NCT05914805) trials, which evaluated patients across 51 study centers in the United States and Europe.

Patients who remained on continuous clascoterone treatment for the full 12 months achieved a statistically significant 239% improvement in Target Area Hair Count (TAHC) compared with patients who received clascoterone for six months and were then switched to vehicle from month 7 to month 12, according to Cosmo.

That’s down slightly from the 252% improvement in TAHC shown for clascoterone versus “vehicle” or placebo in Cosmo’s six-month results, released in December. Cosmo Pharma CEO Giovanni Di Napoli told GEN that the 6- and 12-month results were not directly comparable due to differences in Part 1 and Part 2 of the Phase III placebo-controlled program and the corresponding patient groups being compared.

Part 1 is a double-blind study assessing if clascoterone was effective and safe compared to placebo when applied twice daily for up to six months. Part 2 is a single blind study that measured clascoterone’s long-term safety and efficacy versus placebo for an additional six months in patients who had responded to the study drug in Part 1. During Part 2, participants were re-randomized to receive either clascoterone 5% solution or vehicle solution.

SCALP 1 enrolled 702 patients in the United States, while SCALP 2 enrolled 763 patients in the Unites States as well as Germany and Poland.

Primary outcome measures

Change in vellus TAHC (hair of up to 30 micrometers in diameter) from baseline was the trials’ primary outcome measure, paired with a patient-reported outcome assessing participants’ perception of hair growth improvement.

Additional assessments of the trials included investigator-reviewed global scalp photography and secondary endpoints that included changes in non-vellus TAHC (thicker, pigmented hair >30-40 micrometers in diameter), and changes in subject’s assessment of satisfaction score.

Patients treated with clascoterone for 12 months reported a statistically significant +24.5% relative improvement in treatment satisfaction versus vehicle groups, according to Cosmo. The clascoterone users also reported positive ease of use and product acceptability at month 12—results that according to the company support positive real-world usability and long-term adherence potential for the drug.

Cosmo said it plans to submit its full Phase III dataset for publication in a leading peer-reviewed medical journal and present its findings at future major dermatology congresses.

‘Defining moment’

“These 12-month Phase III results mark a defining moment for clascoterone and for the treatment of male hair loss,” Di Napoli stated. “We are now seeing the combination that matters most: positive long-term safety, statistically significant continued hair growth through one year, and clear evidence that ongoing treatment drives sustained benefit.”

Investors responded to the positive 12-month data by sending Cosmo shares traded on the SIX Swiss Exchange rising 6% on the day of the announcement, from CHF95.50 ($122.69) to CHF101.40 ($130.27) on April 15. Since then shares have fluctuated in the high CHF 90 range, closing Monday at CHF 98.50 ($126.55).

Di Napoli said clascoterone has the potential to emerge as a major new therapeutic option and a highly valuable growth platform for Cosmo by tackling the most common cause of hair loss in men. Androgenetic alopecia, also called male pattern hair loss, affects approximately 40% of men worldwide—including 39% of males in the United States (65 million men).

“We are moving with urgency toward regulatory submissions and commercialization discussions,” Di Napoli added.

Cosmo’s results “likely now enable more advanced partnership discussions, in our view, with detailed presentation of results the next step to fully de-risk the asset,” Benjamin Jackson, equity analyst with Jefferies, wrote April 15 in a research note.

Jackson predicted clascoterone could generate $4 billion in peak-year worldwide sales.

“Our $4 billion WW potential peak sales require just 4% penetration of treated and 6% penetration of untreated men at peak, assuming a capable commercial partner is successfully found,” Jackson added.

Cosmo said it is preparing to submit not only an NDA for clascoterone in the U.S., but a marketing authorization application to the European Medicines Agency.

Clascoterone’s 1% formulation is already FDA-approved and marketed as Winlevi^® for topical treatment of acne vulgaris in patients ages 12 and older.

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Marken UPS Healthcare Precision Logistics doesn’t simply move packages. We manage a complexecosystem of specialized service lines designed to maintain the integrity of priority shipping and lifesaving medications from the point of manufacture to the patient’s bedside. Understanding these services is essential for an appreciation of how modern medicine reaches the global population with its efficacy intact.

Specialty logistics distinguishes itself from general freight through a relentless commitment to technical precision and customized infrastructure. While standard shipping relies on high-volume throughout and routine packaging, specialty logistics demands a bespoke approach to every mile of the journey. This often involves the integration of advanced cold chain innovation to maintain biological integrity or the deployment of specialized technology for visibility of critical materials and equipment in transit.

Beyond the physical hardware, the sector is defined by a rigorous regulatory landscape where practitioners must navigate a complex web of international compliance, hazardous material protocols, and detailed chain-of-custody requirements. In this environment, good enough is never an option. Every variable, from the precise humidity levels of a storage facility to vibration dampening on a pallet shipper, is carefully monitored.

This level of granularity ensures that whether a shipment contains a life-saving pharmaceutical batch or a one-of-a-kind special piece of equipment, it arrives not just on time, but in its exact intended state. Consequently, precision logistics serve as the invisible backbone for industries where the cost of failure far exceeds the cost of transport, necessitating a fusion of engineering, legal expertise, and sophisticated data analytics to manage the inherent risks of moving the world’s most challenging cargo.

Transitioning from the theoretical complexities of the industry to the practical execution of a global supply chain requires an adaptable, expert-led approach. While the challenges of specialized logistics are diverse, ranging from strict thermal requirements to extreme environmental demands, Marken’s operational framework is built on seven distinct pillars of excellence.

Each of these service lines has been engineered to address a specific facet of the logistical puzzle, providing the specialized equipment, certified personnel, and rigorous oversight necessary to mitigate risk. By categorizing our capabilities into these dedicated sectors, we ensure every project receives a novel strategy rather than a one-sizefits- all solution.

These service lines represent more than just transportation or clinical trial categories. They are specialized disciplines derived from decades of work in logistics that allow us to maintain a high rate of success for the world’s most sensitive and high-value cargo. In this eBook, Marken experts share how these precision-driven services ensure the performance, reliability, and success of global supply chains.

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Freudenberg Medical manufactures silicone and thermoplastic elastomer (TPE) tubing for bioprocessing and critical fluid transfer. The company specializes in seamless, overmolded single-use assemblies used in vaccine production, cell cultivation, fluid transfer, and fill-finish operations.

The controlled cleanroom enables customers to receive ready-to-use washed, dried, and gamma sterilized single-use assemblies with the highest product quality, sterility, and process consistency, noted Rudi Gall, vp, global pharma, Freudenberg Medical.

“CleanAssure allows us to support our customers beyond component manufacturing,” he said. “By integrating controlled cleaning and sterilization into our single-use assembly services, we help reduce contamination risk, streamline validation activities, and support a reliable supply for our customers. We can now support customers with their entire component value chain and allow them to focus on their core manufacturing capability.”

Freudenberg’s cleaning process uses ultrapure water and air, operating within ISO 5 conditions. The water is produced using a multi-stage filtration process, resulting in high-purity water specifically suitable for pharmaceutical applications.

Key biopharma industry challenges

Biopharmaceutical manufacturers increasingly rely on single-use systems but face ongoing challenges related to cleaning validation, contamination risk, and production downtime. Customer-managed cleaning processes are often time-intensive, costly, and require additional resources while directly impacting supply reliability, according to Gall.

The company explained that its controlled cleaning environment addresses these challenges by reducing cross-contamination risk through tightly controlled ISO Class 5 processing; alleviating customer cleaning validation burden by delivering assemblies washed and sterilized under cGMP, validated conditions; minimizing production downtime by removing cleaning as a process step; and supporting a consistent, reliable supply of high-quality single-use assemblies.

Freudenberg will be attending INTERPHEX New York, April 21–23, Booth 1673, to exhibit its new products and services.

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A new study from Gladstone Institutes and the University of California, San Francisco (UCSF), changes that. In the study, titled “Systematic Discovery of Pro- and Anti-HIV Host Factors in Primary Human CD4+ T Cells” and published in Cell, researchers report the first genome‑wide map of human genes that either promote or restrict HIV infection in primary human CD4+ T cells, offering a long‑sought blueprint of the host–virus interface.

“HIV has been a global crisis for over 40 years,” said Alex Marson, MD, PhD, director of the Gladstone‑UCSF Institute of Genomic Immunology and senior author of the study. “By studying human T cells, which are the primary target of the virus, we’ve finally mapped the genes—many of which were previously unknown—that influence whether or not they can be infected by HIV.”

With that platform in hand, the researchers performed orthogonal genome‑wide CRISPR activation (CRISPRa) and CRISPR knockout (CRISPRn) screens in CD4+ T cells, systematically testing nearly every human gene. Disrupting genes revealed those HIV depends on, while overactivating genes exposed natural antiviral defenses that HIV normally suppresses. “Over‑activating the genes gave us a wealth of information,” said co–first author Eli Dugan, a PhD candidate in Marson’s lab. “We discovered natural antiviral proteins that were previously invisible because the virus could effectively silence them.”

Across both screens, the team identified hundreds of host factors that shape HIV infection. Among the most striking were two previously unrecognized antiviral proteins: PI16 and PPID (Cyp40). “PI16 interacts with host factors involved in HIV fusion and inhibits viral entry, whereas PPID, a paralog of the proviral cyclophilin CypA, binds capsid and reduces nuclear import of the HIV core,” wrote the authors. Targeted mutagenesis, along with structural modeling and evolutionary analyses, pinpointed residues essential for PPID’s restriction activity, and engineered variants were up to tenfold more potent, according to Dugan.

To test whether these defenses could counter real‑world viral strains, the team collaborated with HIV pioneer Jay Levy, MD, who provided isolates from the early AIDS epidemic. Elevated levels of PI16 or PPID restricted even these aggressive HIV strains.

“This was the first genome‑wide effort to show how human genes affect HIV infection in cells taken directly from human blood samples,” said Nevan Krogan, PhD, director of the HIV Accessory and Regulatory Complexes (HARC) Center. “Our findings could eventually lead to new treatments that help the body’s immune system resist the virus.”

Beyond identifying antiviral factors, the study offers the potential for a powerful new platform for probing HIV latency—the persistent reservoir that evades antiretroviral therapy. “Now, we have the platform to ask the biggest questions in the field,” Rathore said, “and hopefully learn how to eliminate hidden HIV that current drugs can’t reach.”

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Ambitious efforts are on the way that eventually could enable the engineering of entire implantable liver organs. However, the maximum size of laboratory-engineered liver constructs remains limited and cannot yet provide therapeutic benefits for patients. A research team at the Wyss Institute at Harvard University, Boston University, and MIT has now approached this important problem from a different angle.

“We asked if it would be possible to first implant a small-scale liver construct and then drive it to expand in the body following its engraftment,” said Christopher Chen, MD, PhD, a Wyss Institute core faculty member and the William Fairfield Warren Distinguished professor of biomedical engineering and director of the Biological Design Center at Boston University. “A sufficiently grown, functional ‘satellite liver’ could immediately relieve the metabolic burden in a damaged liver and help bridge the time until a transplant becomes available.”

Chen co-led the research together with associate faculty member Sangeeta Bhatia, MD, PhD, who is the John J. and Dorothy Wilson Professor of Health Sciences and Technology and of Electrical Engineering and Computer Science at the Koch Institute for Integrative Cancer Research at MIT, and a Howard Hughes Medical Institute investigator. Chen is also a leader of the Wyss Institute’s 3D Organ Engineering Initiative, and team lead of the recently awarded ARPA-H PRINT-supported ImPLANT project, which focuses on whole organ liver engineering at the Wyss and collaborating institutions.

The project, spearheaded by Amy Stoddard, PhD, (MIT ’25), who developed the approach in her doctoral research and then as a postdoctoral fellow, integrates tissue engineering and synthetic biology tools in a genetic strategy the team has named “bioengineered on-demand outgrowth via synthetic biology triggering,” or BOOST. By specifically rewiring the gene expression of primary liver hepatocytes and supportive fibroblast cells, the scientists were able to effectively switch on a tissue growth program in small, engineered liver constructs after their implantation into mice.

“Using engineered liver tissue as a proof-of-concept application, we integrated synthetic biology and tissue engineering tools to build liver tissues that can be expanded on-demand after implantation in vivo,” the team reported in their published paper in Science Advances, which is titled “Synthetic control of implanted engineered liver tissue growth.” In the paper they concluded “In this study, we define the first steps toward an unconventional approach to cell therapy scale-up: engineering a small construct and then inducing it to grow in situ … “This strategy, which we have named BOOST, could provide several key advantages, including circumventing the need for large quantities of cellular raw materials and bypassing the formidable challenge of generating a rapidly perfusable construct that can survive the engraftment period.”

The authors wrote, “Organ transplant is currently the only curative treatment for patients with end-stage organ failure, yet this therapy is inaccessible to many due to the paucity of organs available for transplant.” And while significant progress has been made in the field of engineering tissue-based cell therapies that could represent alternatives, or bridges to transplant, they acknowledge, “… scaling of these constructs to sizes of therapeutic relevance remains a barrier to clinical translation.”

In order to address current challenges associated with fabrication, Chen and colleagues looked at the problem from different angle, asking whether it would be possible to first implant a small-scale construct and then trigger it to expand in situ, after its engraftment into the host.

To be able to induce growth of an implanted small liver constructs in situ within a recipient’s body the researchers first needed to identify the relevant cues that would allow them to do so. “A key first step toward this method of in situ scale-up would be the successful control of cellular growth within the engineered construct after engraftment,” they wrote. Since liver growth is known to be regulated by soluble growth factors (GFs), Stoddard screened a collection of candidate factors to identify those that, when added to cultured human primary hepatocyte cells (HEPs), had the strongest growth-inducing effects.

The team homed in on a protein, YAP, that senses mechanical signals, and which was known to move from cells’ cytosol to their nucleus in low-density conditions to help express genes involved in cell proliferation. However, in high-density conditions when cells are compressed, YAP is degraded in the cytosol, which prevents the activation of those target genes and restricts proliferation.

“Importantly, when we overexpressed a non-degradable version of YAP in HEPs, which also reaches the nucleus in high-density conditions to partake in gene regulation, we successfully overrode this density checkpoint in HEPs,” Stoddard said. “Interestingly, we found that HEPs needed to be stimulated with both YAP and GFs in order to grow in densely packed 3D liver tissues.”

Toward the goal of safely inducing and controlling HEP proliferation in a living organism, and eventually human patients, the researchers deployed synthetic biology tools to locally install control of these signaling pathways in HEPs and fibroblast cells within the engineered 3D liver tissues themselves. “We set out to engineer a synthetic biology toolkit capable of locally modulating growth factor and YAP signaling within engineered liver tissue, enabling on-demand control of proliferation even after implantation,” they noted.

The team engineered fibroblast cell lines that each secreted one of the four GFs, and HEPs that expressed the non-degradable YAP protein. And they made the expression of all proteins doxycycline (DOX)-inducible. They determined in time course experiments that a continuous seven-day treatment with DOX led 3D liver tissue composed of engineered cells to robustly expand in size and cell numbers in the culture dish. On DOX removal the HEPs reverted back to a non-proliferating state.

However, Stoddard noted, “… when we compared the gene expression of single cells in BOOST-engineered, DOX-induced 3D liver tissue to that of cells in non-engineered or BOOST-engineered, non-induced 3D liver tissue, we noticed that the expansion came with a trade-off: high proliferation rates went hand in hand with a less functional HEP state. While we believe this is a natural trade-off seen in a wide variety of biological settings, we hope to be able to address this in the future, recognizing that the liver also has native re-functionalization signals to harness.”

The litmus test for BOOST-engineered growth in 3D liver tissues was to see whether they would similarly expand following their implantation into living mice that were systemically treated with DOX for the same seven-day duration. Experiments showed that the implanted tissue exhibited a striking 500% increase in proliferation with a doubling of the engineered HEPs alone, and was vascularized to accommodate the metabolic demands of the expanded tissue. The tissue implants were also well tolerated by the mice, with no signs of fibrosis due to invading immune cells and fibroblast inflammation, or of tumor growth.

“These results were particularly exciting to us,” said Stoddard. “Prior to our work, injury to the host liver has always been required to trigger hepatocyte engraftment and proliferation. Here we were able to relieve this dependence, and trigger on-demand growth of implanted liver tissue in a completely healthy host.”

In the future, the team will explore the capacity of BOOSTed liver tissue to rescue the host in the setting of liver injury. “Our BOOST strategy lays the foundation for a future when solid organ cell therapies can be controlled non-surgically according to the needs of patients and their physicians,” Bhatia noted. “Beyond treating liver disease, the premise of BOOST could be applied to other engineered tissue therapeutics that are similarly constrained by challenges associated with tissue scale-up, such as engineered heart or pancreatic tissue to address serious diseases.”

In their paper the authors concluded, “… this work serves as an exciting proof-of-concept demonstration that scale-up of tissues via growth could be possible … Together, this work helps lay the foundations for a future of ‘smart’ tissue therapeutics that can be scaled to a patient’s needs and thereby offer treatment for numerous, previously incurable, diseases.”

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Kelonia’s lead program KLN-1010 is a one-time intravenous gene therapy designed to generate anti-B-cell maturation antigen (BCMA) CAR T cells, targeting the BCMA protein expressed on the surface of multiple myeloma cells.

In December at the American Society of Hematology (ASH) 2025 Annual Meeting, Kelonia presented positive early clinical data for KLN-1010 from the Phase I inMMyCAR^TM trial (NCT07075185). The data showed the CAR T therapy to have 100% minimal residual disease (MRD)-negative response rate across four patients, all of whom remained in response through the longest follow up of five months.

Those and other results, according to the company, provided initial clinical validation of KLN-1010 and demonstrated promising tolerability. In January, Kelonia won FDA clearance for an investigational new drug (IND) application for KLN-1010, enabling the trial to expand from Australia into multiple clinical sites across the United States.

“The early clinical data for KLN-1010 are highly encouraging, both as a potential step forward for patients with multiple myeloma and as proof of concept for Kelonia’s platform,” Jacob Van Naarden, executive vice president and president of Lilly Oncology and head of corporate business development, said in a statement.

Investors appeared more sanguine about the Kelonia acquisition as Lilly shares were all but flat in early Monday trading as of 11 a.m. ET, to $927.16 from Friday’s close of $927.03. Kelonia is privately held.

KLN-1010 applies the company’s in vivo gene placement system (iGPS^®), which uses engineered lentiviral-based particles designed to efficiently and selectively enter T-cells inside the body, enabling a patient’s own body to generate CAR T therapies designed to treat underlying disease.

Lilly and Kelonia reason that KLN-1010 could transform treatment of multiple myeloma by eliminating challenges associated with both ex vivo patient-specific cell therapy manufacturing, and pre-administration chemotherapy.

“Autologous CAR T therapies have meaningfully improved outcomes for patients with various cancers, but significant manufacturing, safety, and access barriers mean that only a fraction of eligible patients actually receive them,” Van Naarden added. “Kelonia’s in vivo platform has the potential to change that by delivering rapid, durable responses in a far simpler, off-the-shelf format.”

Kelonia marks Eli Lilly’s fourth announced acquisition of a smaller biotech this year:

• In March, Lilly committed up to $7.8 billion to acquire Centessa Therapeutics, a developer of sleep disorder drugs.
• A month earlier, Lilly announced plans to buy out circular RNA cell therapy developer Orna Therapeutics for up to $2.4 billion, targeting advancements in cell therapy.
• And in January, Lilly inked a $1.2 billion acquisition of Ventyx Biosciences, an NLRP3-targeting oral drug developer focused on inflammatory diseases.

Behind the deals

Behind all the deals is the pharma giant’s desire to capitalize on the billions of dollars it is generating from sales of its obesity and diabetes drugs based on glucagon-like peptide 1 (GLP-1) receptor analysts alone or in tandem with a glucose-dependent insulinotropic polypeptide (GIP). Lilly markets tirzepatide, a GLP-1/GIP dual agonist, in obesity as Zepbound^® ($13.542 billion in 2025 sales) and in diabetes as Mounjaro^® ($22.965 billion).

Lilly stands to generate even more in obesity-related sales in coming years once it brings to market its oral obesity drug Foundayo (orforglipron), a small molecule GLP-1 receptor agonist—though analysts predict the drug’s 2026 sales will likely be lower than once expected because of the price war Foundayo faces competing head to head with Lilly’s arch-rival in obesity drugs, Novo Nordisk. In December, Novo Nordisk got a jump on Lilly when the Danish biotech giant won FDA approval for oral Wegovy^® (semaglutide), a once-daily 25 mg GLP-1 receptor agonist tablet indicated for chronic weight management.

A Lilly buyout of Kelonia could compel Johnson & Johnson to take a closer look at acquiring Legend Biotech, Kostas Biliouris, PhD, a managing director on the biotechnology research team of Oppenheimer, wrote Sunday in a research note. He cited the fact J&J’s Janssen Biotech successfully partnered with Legend to develop Carvykti^® (ciltacabtagene autoleucel), a B-cell maturation antigen (BCMA)-directed CAR T-cell therapy indicated for adults with relapsed or refractory multiple myeloma who have received at least one prior line of therapy. Carvykti generated $1.877 billion in sales last year, up nearly double (96%) from $963 million in 2024.

Also, Biliouris cited the presence in Legend’s pipeline of LUCAR-G39D, a clinical in vivo CAR T program designed to treat B-cell non-Hodgkin’s lymphoma by targeting CD19xCD20. LUCAR-G39D showed positive first-in-human safety and efficacy data from a Phase I trial (NCT06395870) at ASH last December.

“We believe in vivo CAR T technology has strong potential, as treatment process is fast and circumvents the need for lymphodepletion, but think it will likely take ~6-8years before safety/durability questions are addressed, and regulatory approval is granted,” Biliouris predicted.

Lilly has agreed to acquire Kelonia for $3.25 billion upfront plus up to $3.75 billion in future payments tied to achieving specified clinical, regulatory, and commercial milestones. The acquisition deal is subject to regulatory approvals and other customary closing conditions, and is expected to close in the second half of 2026.

Upon closing, Lilly said, it will determine how to account for the transaction in accordance with Generally Accepted Accounting Principles (GAAP), then reflect the deal in future financial results and financial guidance.

“Kelonia’s leadership in advancing the immense promise of in vivo cell therapy is unmatched, extending its reach and impact beyond the traditional boundaries of personalized medicine,” Kelonia CEO Kevin Friedman, PhD, stated. “We have demonstrated the ability to achieve deep multiple myeloma remissions with significantly reduced complexity and cost relative to ex vivo CAR T-cell approaches.”

“In combination with Lilly’s strengths, our in vivo iGPS platform is positioned to broaden the reach of cell therapy beyond the current CAR T landscape in hematologic malignancies and to transform treatment across a far wider range of cancers and other serious diseases,” Friedman added.

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“Our findings suggest that DNA-based approaches can help identify where a cancer may have started, even when the original tumor is not visible,” said Marco A. De Velasco, PhD, a faculty member in the department of genome biology at Kindai University in Japan.  

CUP are metastatic malignancies in which the primary cancer site could not be identified. These cancers are often associated with poorer outcomes, as patients are typically treated with broad, nonspecific chemotherapy regimens rather than therapies targeted to a specific cancer type. 

Approximately only 15-20% of patients with CUP show features that allow site-specific therapies. Patients receiving site-directed therapy can survive up to 24 months, compared with six to nine months for those receiving standard treatment. 

Patterns in tumor biology, such as gene activity or chemical modifications to DNA, can differ between cancer types and persist even after the cancer has spread and guide development of these therapies. While some methods have shown promise, they have yet to demonstrate clear survival benefits in clinical trials. 

The model was developed using methylation data from nearly 7,500 patients with 21 different cancer types obtained from The Cancer Genome Atlas Program and other public datasets. Using machine learning, the researchers identified CpG methylation and built methylation profiles that were associated with different tumor types. 

Del Velasco emphasized that the study achieved high accuracy in predicting the origin of diverse cancer types using a small subset of DNA markers, about 1,000 CpG regions selected from hundreds of thousands across the genome. “This is important because it shows that we can simplify complex molecular data while still maintaining strong predictive performance,” he said. 

As a limitation, the model was developed using cancers with known origins, rather than true CUP. Testing in CUP patients is important to understand how well the model performs in clinical settings. Additionally, not all tumors are easily accessible for genetic testing, particularly tumors in advanced stage. Looking ahead, the authors aim to adapt and evaluate the model using blood-based biopsy to analyze circulating tumor DNA instead of relying on DNA from tissue samples. 

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