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Cell & Gene Therapy Review

Cell & Gene Therapy Review

@cellgenetherapyreview.com

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https://www.cellgenetherapyreview.com/ The #CGT industry’s comprehensive information source for #CellTherapy and #GeneTherapy business and technology

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Cell & Gene Therapy Review @cellgenetherapyreview.com · 2d

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. 🧪🧬⑂

Galapagos to wind down cell therapy business

According to the company, the decision follows a comprehensive review of strategic alternatives, including a potential divestiture

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Cell & Gene Therapy Review @cellgenetherapyreview.com · 26d

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.🧪🧬⑂

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...

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Sep 3

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 🧪🧬⑂

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...

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jul 31

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. 🧪🧬⑂

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...

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jul 30

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. 🧪🧬⑂

CBER head Vinay Prasad resigns amid controversy

Prasad, tangled in a public dispute with Sarepta, has left the FDA after less than three months as director of CBER.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jul 9

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. 🧪🧬⑂

New center to offer personalized 'CRISPR cures' for kids with rare diseases

The Center for Pediatric CRISPR Cures will be funded by a $20 million grant from the Chan Zuckerberg Initiative.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jul 3

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. 🧪🧬⑂

Rinri gets UK nod to launch cell therapy hearing loss trial

Proof-of-concept data from the phase 1/2a trial of the first-in-class optic neural progenitor cell therapy is expected within 12 months of trial initiation.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jul 1

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. 🧪🧬⑂

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...

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jun 25

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. 🧪🧬⑂

Vertex shares updated data for islet cell therapy in diabetes

All patients who received a full dose of zimislecel achieved ADA-recommended target HbA1c levels and 10/12 patients were insulin free.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jun 23

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. 🧪🧬⑂

FDA to halt trials that send Americans’ cells to labs in “hostile” countries

The agency will immediately review clinical trials that involve sending Americans' living cells to China and other “hostile” countries for genetic engineering.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jun 17

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. 🧪🧬⑂

Lilly to buy gene editing biotech Verve for $1.3B

Lilly will pick up the biotech’s pipeline of gene editing meds designed to address the drivers of atherosclerotic cardiovascular disease through one-time treatments.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jun 16

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. 🧪🧬⑂

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...

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Jun 9

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. 🧪🧬⑂

Trump administration shares updated NIH budget details

The White House has released an updated 2026 budget proposal for the HHS, outlining more detailed reorganization plans, including NIH cuts.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · May 27

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).

Industry groups call for 10-year halt on heritable human genome editing

ISCT, ARM and ASGCT have released a joint statement reaffirming the need for responsible oversight of genetic technologies that could alter the human germline.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · May 23

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). 🧪🧬⑂

BrainStorm gets FDA green light for phase 3 trial of ALS cell therapy

The FDA has cleared BrainStorm to initiate its phase 3b clinical trial of its autologous MSC-NTF cell therapy, NurOwn.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · May 22

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. 🧪🧬⑂

Atsena shares positive early data in ocular gene therapy trial

Atsena announced positive results from Part A of a phase 1/2 clinical trial evaluating subretinal injection of ATSN-201for X-linked retinoschisis.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · May 21

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

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...

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Cell & Gene Therapy Review @cellgenetherapyreview.com · May 19

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 [email protected] and be featured in our event recap.

Cell & Gene Therapy Review @cellgenetherapyreview.com · May 19

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

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...

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Cell & Gene Therapy Review @cellgenetherapyreview.com · May 6

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

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...

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Cell & Gene Therapy Review @cellgenetherapyreview.com · May 2

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

Chengdu Origen, Vanotech dose first patient in wet AMD gene therapy trial

The companies have dosed the first wet age-related macular degeneration patient in a phase 1 trial evaluating treatment with an AAV gene therapy candidate.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · May 1

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

FDA approves Abeona gene therapy for rare skin disease

Zevaskyn is the only FDA-approved product to treat recessive dystrophic epidermolysis bullosa wounds with a single application.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Apr 30

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. 🧪🧬⑂

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...

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Apr 28

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

Ferring expands gene therapy production to U.S. site

The U.S. FDA has approved Ferring’s drug product manufacturing hub in Parsippany, New Jersey, for its intravesical non-replicating gene therapy, Adstiladrin.

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Cell & Gene Therapy Review @cellgenetherapyreview.com · Apr 25

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

Caribou trims staff, narrows pipeline to two oncology CAR-Ts

The company announced a pipeline prioritization with workforce and cost reduction initiatives to focus resources on two oncology clinical programs.

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