The documents cover topics including efficient trial design, expedited review programs, and postapproval data collection.
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In observance of Rare Cancer Day, held annually on September 30, catch up on the past few months’ news related to cell therapy for such indications.
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In observance of World Heart Day, held annually on September 29, we took a look back at news in cell/gene therapy for cardiovascular disease from the past few months.
MVX-220 is intended to provide a functional copy of UBE3A, the disease-targeted gene, to the neurons.
Review top news and interview highlights from the week ending September 26, 2025.
SAR446268 has previously received orphan drug designations from the FDA and European Medicines Agency.
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The study also met a key secondary end point.
Jainu Jogani, the cofounder of Child’s Cure Genetic Research, discussed his daughter’s rare genetic disease and the need for new treatment options.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Part B of LIGHTHOUSE enrolled 9 patients and is expected to enroll 3 pediatric patients.
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The cofounder of Child’s Cure Genetic Research discussed his daughter’s rare genetic disease and the need for new treatment options.
In honor of Duchenne Action Month and World Duchenne Day, held every year in September, CGTLive is taking a closer look at this ongoing study.
Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, discussed a trend of physicians and scientists working on models to make gene therapy products commercially viable.
The director of the Mass General Brigham Gene and Cell Therapy Institute discussed a trend of doctors and scientists working on models to make gene therapy products financially viable.
Review top news and interview highlights from the week ending September 19, 2025.
The director of the Mass General Brigham Gene and Cell Therapy Institute discussed innovations in viral vectors, CAR-T, and more.
The trial, which is taking place in the United States, is expected to enroll 40 patients in total.
Daniel Galbraith, PhD, the chief scientific officer of Solvias, discussed potential uses for the company’s platform.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
SAR402663 comprises a onetime intravitreal treatment and is intended to inhibit VEGF by providing a gene for soluble FLT01.
The chief scientific officer of Solvias discussed potential uses for the company’s platform.
With regard to safety, DTX401’s safety profile was characterized as “acceptable and expected.”
In episode 6 of ImmunoLogic, Stephen Schoenberger, PhD, discussed his research on customized cancer vaccines.
For World Lymphoma Awareness Day, held annually on September 15, take a look at the stories that have stood out in lymphoma cell therapy in recent months.
In observance of Duchenne Action Month and World Duchenne Day, held annually in September, we took a look back at recent news in gene and cell therapy for DMD.
Review top news and interview highlights from the week ending September 12, 2025.
Capsida has made the move to pause the trial voluntarily.
The company specifically highlighted that one patient who was treated in the trial’s first cohort is still alive at 28 months.
According to REGENXBIO, HS D2S6 is “a key biomarker of MPS II brain disease that is reasonably likely to predict clinical benefit.”
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
