Cell & Gene Therapy Review
@cellgenetherapyreview.com
https://www.cellgenetherapyreview.com/
The #CGT industry’s comprehensive information source for #CellTherapy and #GeneTherapy business and technology
The #CGT industry’s comprehensive information source for #CellTherapy and #GeneTherapy business and technology
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. 🧪🧬⑂
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
www.cellgenetherapyreview.com
October 23, 2025 at 2:17 PM
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. 🧪🧬⑂
According to the company, the decision follows a comprehensive review of alternatives, including a potential divestiture. 🧪🧬⑂
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...
www.cellgenetherapyreview.com
September 29, 2025 at 7:11 PM
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.🧪🧬⑂
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...
www.cellgenetherapyreview.com
September 3, 2025 at 5:59 PM
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 🧪🧬⑂
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. 🧪🧬⑂
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...
www.cellgenetherapyreview.com
July 31, 2025 at 5:15 PM
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.
www.cellgenetherapyreview.com
July 30, 2025 at 5:41 PM
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.
www.cellgenetherapyreview.com
July 9, 2025 at 5:42 PM
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. 🧪🧬⑂
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.
www.cellgenetherapyreview.com
July 3, 2025 at 2:33 PM
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. 🧪🧬⑂
Clinical proof-of-concept data from the phase trial is expected within 12 months of initiation. 🧪🧬⑂
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...
www.cellgenetherapyreview.com
July 1, 2025 at 5:29 PM
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. 🧪🧬⑂
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.
www.cellgenetherapyreview.com
June 25, 2025 at 1:18 PM
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. 🧪🧬⑂
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.
www.cellgenetherapyreview.com
June 23, 2025 at 2:03 PM
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.
www.cellgenetherapyreview.com
June 17, 2025 at 3:20 PM
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. 🧪🧬⑂
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...
www.cellgenetherapyreview.com
June 16, 2025 at 7:15 PM
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. 🧪🧬⑂
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.
www.cellgenetherapyreview.com
June 9, 2025 at 5:15 PM
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).
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.
www.cellgenetherapyreview.com
May 27, 2025 at 2:35 PM
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). 🧪🧬⑂
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.
www.cellgenetherapyreview.com
May 23, 2025 at 2:51 PM
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. 🧪🧬⑂
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.
www.cellgenetherapyreview.com
May 22, 2025 at 6:00 PM
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. 🧪🧬⑂
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...
www.cellgenetherapyreview.com
May 21, 2025 at 5:05 PM
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.
May 19, 2025 at 7:24 PM
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.
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...
www.cellgenetherapyreview.com
May 19, 2025 at 3:46 PM
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
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
#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...
www.cellgenetherapyreview.com
May 6, 2025 at 5:20 PM
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
#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
#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.
www.cellgenetherapyreview.com
May 2, 2025 at 5:22 PM
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
#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
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.
www.cellgenetherapyreview.com
May 1, 2025 at 5:16 PM
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
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...
www.cellgenetherapyreview.com
April 30, 2025 at 4:25 PM
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
🧪🧬⑂ #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.
www.cellgenetherapyreview.com
April 28, 2025 at 3:59 PM
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
🧪🧬⑂ #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
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.
www.cellgenetherapyreview.com
April 25, 2025 at 5:31 PM
Caribou Biosciences announced a strategic pipeline prioritization with workforce and cost reduction initiatives to focus resources on two lead oncology clinical programs.🧪🧬⑂ #CellTherapy #SciComm