After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.

There are currently no approved disease-modifying therapies for MPS IIIA.

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

US biotech firm Fractyl Health is developing Rejuva, an experimental gene therapy that may mimic GLP-1 drugs for years after ...

Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

A U.K. center of excellence for gene and cell therapies is promoting the benefits of digital tools to aid the manufacturing.

Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotypeBLA resubmitted to U.S. FDA in January 2026; ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other parts of the brain. A new preclinical study has identified a gene therapy ap ...

ROCKVILLE, Md., July 10, 2025 /PRNewswire/ -- REGENXBIO Inc. today announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the C-terminal (CT) ...

Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...