A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to ...

Vitamin D is not only an essential nutrient, but also the precursor of the hormone calcitriol, indispensable for health. It ...

Sarepta Therapeutics Inc. said another patient has died from acute liver failure after receiving one of its gene therapies, putting additional pressure on the biotech company after the recent deaths ...

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

Sarepta Therapeutics, Inc. faces setbacks with Elevidys, safety issues, and workforce cuts, raising concerns about its ...

The FDA granted fast track designation to SAR446597, an intravitreal gene therapy for the treatment of geographic atrophy due to age-related macular degeneration, according to a press release from ...

Caribou Biosciences' allogeneic CAR T-cell therapies show promising early efficacy and safety, but require clinical ...

AAVB-039 advances gene therapy for Stargardt disease, aiming for innovative treatments for inherited retinal disorders.

Sarepta Therapeutics appears to have right-sized itself after laying off over a third of its staff, announcing a significant ...

Want a bespoke baby? Check the beauty, brains, or brawn boxes on the embryo order form. Genetic tinkering is no longer science fiction—it’s a market without legal guidelines or societal buy-in. But ...

The US Food and Drug Administration (FDA) recently granted fast track designation to Sanofi’s SAR446597 for the treatment of geographic atrophy (GA). 1 ...

Discover the current pipeline candidates advancing through the clinic for the treatment of sickle cell disease.