Gene Therapy (AAVrh74-MHCK7-βSG)

About Sarepta Therapeutics

Sarepta Therapeutics is a pioneering, commercial-stage biotech focused on developing and commercializing precision genetic medicines for devastating rare diseases, primarily Duchenne muscular dystrophy. The company has multiple FDA-approved exon-skipping therapies on the market and is advancing a robust pipeline that includes a commercial gene therapy and other candidates for DMD and limb-girdle muscular dystrophy (LGMD). With a hybrid manufacturing strategy and a relentless focus on following the science, Sarepta aims to reclaim futures for patients impacted by rare genetic disorders.

Therapeutic Areas