Cell & Gene Therapy Review

Galapagos announced its intention to wind down its #CellTherapy business and “pursue new transformational business development transactions” with its available cash.

According to the company, the decision follows a comprehensive review of alternatives, including a potential divestiture. 🧪🧬⑂

How digital can ease the burden of cell and gene therapy long-term follow-up Because CGTs are designed to have lasting and lifelong effects, regulatory bodies often mandate 15-year, long-term follow-up. Fortunately, collaborative groups are creating resources and recommendatio...

Because #CGTs are designed to have lasting and lifelong effects, regulatory bodies often mandate 15-year, long-term follow-up. LTFU allows the industry to uncover potential delayed or rare side effects while measuring the durability of treatment, making the treatment better understood and safer.🧪🧬⑂

Navigating cell therapy's maze of risks Biopharma companies advancing cell therapies must confront a range of key considerations and risks. A strategic, collaborative approach can accelerate development and ensure patients reliably access s...

Biopharma companies advancing cell therapies must confront a range of key considerations and risks. A strategic, collaborative approach can accelerate development and ensure patients reliably access safe and effective cell therapy products 🧪🧬⑂

MSCs rising With MSCs back in the spotlight and a pipeline of mid- and late-stage candidates pushing toward regulatory submission, what looms now is not a question of whether MSCs can deliver, but the challenge o...

The FDA’s approval of Mesoblast’s Ryoncil is more than a regulatory milestone — it’s a turning point for #MSC therapies and the broader #CGT field. 🧪🧬⑂

Vinay Prasad has left the #FDA after less than three months as director of #CBER, a government spokesperson confirmed to media outlets late Tuesday. In addition to regulating #vaccines and #CGTs, Prasad was also the FDA's chief medical and scientific officer. 🧪🧬⑂

The Chan Zuckerberg Initiative and Innovative Genomics Institute announced the funding of the new 'Center for Pediatric CRISPR Cures' that will use CRISPR-based editing technology to advance cures for severe pediatric genetic diseases and will bridge #CRISPR design and testing with treatment. 🧪🧬⑂

Rinri Therapeutic announced the approval of its #ClinicalTrial application by UK drug regulator MHRA, greenlighting the first-in-human trial for the company’s regenerative #CellTherapy, Rincell-1.

Clinical proof-of-concept data from the phase trial is expected within 12 months of initiation. 🧪🧬⑂

Unlocking the potential of unconventional T cells The cutting edge of cancer research is now expanding beyond conventional T cells to a less-explored, unconventional, unique subset of T cell: mucosal-associated invariant T (MAIT) cells. Engineered MA...

The cutting edge of #CancerResearch is now expanding beyond conventional #Tcells to a less-explored, unconventional, unique subset of T cell: mucosal-associated invariant T (MAIT) cells. Engineered #MAITcells may stand to change future #AllogeneicTreatments for solid tumors. 🧪🧬⑂

Vertex shared new data from its FORWARD-101 trial of zimislecel, a #StemCell derived islet #CellTherapy for type 1 #diabetes. Among 12 patients followed for at least a year, results show consistent, durable benefits and support the therapy’s transformative potential. 🧪🧬⑂

The #FDA announced an immediate review of new clinical trials involving sending U.S. citizens’ living #cells to China and other “hostile” countries for genetic engineering and infusion back into U.S. patients.

Beyond specifically mentioning China, a list of hostile countries was not provided. 🧪🧬⑂

Eli Lilly will acquire Verve Therapeutics, picking up the #biotech ’s pipeline of #GeneEditing medicines designed to address the drivers of atherosclerotic cardiovascular disease ( #ASCVD ) through one-time #treatments. 🧪🧬⑂

Unpacking cell and gene therapy progress Against a backdrop of historic charm, bold visions for the future of medicine were exchanged as attendees from all over the globe showed up in force to learn, present, collaborate and reconnect on all...

Against a backdrop of historic charm, bold visions for the future of medicine were exchanged as attendees from all over the globe showed up in force to learn, present, collaborate and reconnect on all aspects of #CGT at #ISCT2025 and #ASGCT2025. 🧪🧬⑂

The White House has released an updated 2026 budget proposal for the Department of Health and Human Services, outlining more detailed reorganization plans, including budget cuts and restructuring of the National Institutes of Health. 🧪🧬⑂

Three #CGT industry groups — the International Society for Cell & Gene Therapy, the Alliance for Regenerative Medicine, and the American Society of Gene & Cell Therapy — have released a joint statement calling for a 10-year international moratorium on heritable human genome editing (HHGE).

BrainStorm Cell Therapeutics announced that the U.S. FDA has cleared the company to initiate its phase 3b clinical trial of its autologous MSC-NTF #CellTherapy, NurOwn, for the treatment of amyotrophic lateral sclerosis (ALS). 🧪🧬⑂

Atsena Therapeutics has announced positive clinical data results from Part A of the LIGHTHOUSE study, a phase 1/2 clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS), a genetic condition that leads to blindness. 🧪🧬⑂

Overcoming CGT starting material challenges Much of the focus for making CGTs accessible and commercially viable has looked downstream at manufacturing, logistics, geography, centralization, and even intellectual property. However, the vast maj...

Efforts to make CGTs accessible and commercially viable focus on downstream issues like manufacturing and logistics. But most ex vivo #AdvancedTherapies begin with blood — and the future of #CGT depends on improving how we get what we need from patients’ and donors’ #blood. 🧪🧬⑂ #SciComm #BioTech

Attend an impactful session? Come across an amazing poster presentation or new technology on the show floor? Please share your top takeaway from #ASGCT2025 or #ISCT2025 with us! Send us your insights (up to 200 words) at klanghauser@comparenetworks.com and be featured in our event recap.

Shooting for immune While it is still early days for CGTs in autoimmune disease, the field is actively pushing its own limits, working to translate scientific excitement into better, more accessible products. Not only co...

While it is still early days for CGTs in #autoimmunedisease, the field is actively pushing its own limits, working to translate scientific excitement into better, more accessible products. Not only could this lift #celltherapies to new heights, but it could redefine them entirely. 🧪🧬⑂ #SciComm

Above the noise: CGT developers focus on access to better treatments amid immunology boom The urgency of unmet need has not evaded the CGT sector, which has executed a ‘strategic pivot’ into autoimmune disease. Developers with B-cell targeting therapies originally designed to detect and ki...

From efforts to overhaul manufacturing for autologous therapies to explorations of allogeneic platforms and novel therapeutic designs, the CGT field is raising the bar. 🧪🧬⑂

#SciComm #CGT #PharmaNews

Chengdu Origen and Vanotech, both subsidiaries of Chengdu Kanghong Pharmaceutical Group, announced the first wet age-related macular degeneration patient dosed in a phase 1 multi-center clinical trial evaluating treatment with an #AAV gene therapy candidate. 🧪🧬⑂

#SciComm #GeneTherapy #PharmaNews

The U.S. FDA has approved Abeona Therapeutics’ autologous cell-based #GeneTherapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), a serious and debilitating genetic skin disease. 🧪🧬⑂ #SciComm

From bispecifics and ADCs to better reagents Advances have allowed for the creation of bispecific, ‘Fc-silenced,’ and other specialized antibody reagents tailored to specific research needs. These engineered antibodies are deepening our understa...

Advances have enabled the creation of bispecific, ‘Fc-silenced,’ and other specialized antibodies tailored to specific research needs. These engineered antibodies deepen our understanding of complex biological systems and accelerate research that informs next-generation CGT development. 🧪🧬⑂

The U.S. FDA has approved Ferring Pharmaceuticals’ drug product manufacturing hub in Parsippany, New Jersey, for its intravesical non-replicating gene therapy, Adstiladrin.
🧪🧬⑂ #GeneTherapy #SciComm

Caribou Biosciences announced a strategic pipeline prioritization with workforce and cost reduction initiatives to focus resources on two lead oncology clinical programs.🧪🧬⑂ #CellTherapy #SciComm

March Biosciences has dosed its first patient in a phase 2 trial evaluating its first-in-class CD5-targeted CAR-T cell therapy for patients with relapsed or refractory CD5-positive T-cell lymphoma. 🧪🧬⑂

#CellTherapy #SciComm

Bayer’s BlueRock has published 18-month data from its phase 1 clinical trial for its stem cell therapy, bemdaneprocel, for Parkinson’s disease. 🧪🧬⑂

Bemdaneprocel is the first investigational allogeneic pluripotent stem cell-derived #CellTherapy for Parkinson’s.

#SciComm #PharmaNews

CERo Therapeutics announced that it has entered into a securities purchase agreement for the issuance and sale of securities under a new convertible preferred stock transaction. The gross proceeds from the offering are expected to be up to $8 million.🧪🧬⑂

#SciComm #PharmaNews

The U.S. #FDA has granted breakthrough therapy designation to Amsterdam-based uniQure’s gene therapy candidate, AMT-130, for the treatment of Huntington’s disease. The designation is supported by clinical data from ongoing phase 1/2 trials.🧪🧬⑂

#GeneTherapy #SciComm #PharmaNews #GeneEditing

Thermo Fisher has announced the grand opening of its Advanced Therapies Collaboration Center (ATxCC) in Carlsbad, California. The 6,000-square-foot facility will offer clinical and commercial process dev support to help #CellTherapy devs transition from research-scale to #CGMP manufacturing.🧪🧬⑂