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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.

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.

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.

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.