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.

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 ...

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

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 ...

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

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 ...

Study supports favorable safety and tolerability profile and clinically meaningful functional and structural benefits in Stargardt disease ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

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) ...

Gene therapy for weight loss is being explored as a potential game-changer, with therapy aiming to program the body to ...