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 ...

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 ...

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.

An expert discusses how high-cost gene therapies require evidence standards including Phase 3 trial data showing complete response rates and durability, real-world evidence and health economic ...

The favorable market reaction indicates investor relief at management’s swift measures to rectify the company's worsening ...