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.

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 ...

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

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 ...

NEW ORLEANS — New data from a first-in-human trial of a CRISPR-based gene therapy for cholesterol disorders are giving lipids experts reason for optimism about the approach for treating cardiovascular ...

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

Ocugen has announced the publication of positive results from its phase 1 GARDian1 trial of OCU410ST, a gene therapy for ...

Doctors have delivered gene therapy during heart bypass surgery for the first time, aiming to strengthen blood vessels ...

Two of Opus's seven gene therapy assets are in clinical trials, with additional programs expected to enter the clinic.

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