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

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.

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...

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

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

The gene-therapy company is restructuring, laying off 36% of its workforce and making changes to its executive team.

Sarepta Therapeutics said it will eliminate 36% of its workforce—approximately 500 jobs—in a restructuring that follows the ...

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

The company is also pausing research for several treatments it has been developing for another form of muscular dystrophy.

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 ...