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 ...

Sarepta Therapeutics is facing a challenging period after the deaths of two patients who received its Columbus-invented gene ...

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 ...

Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) surged 15% after receiving a key regulatory boost from the U.S. Food and Drug ...

Sarepta stock surged after the pharma company said it would lay off a third of its workforce and add a black-box warning to ...

Controlling the activity of specific genes in the laboratory through the editing of their epigenetic marks is now a reality ...

Eight children in the U.K. have been spared from devastating genetic diseases thanks to a new three-person in vitro fertilization technique.