bidridistrogene fuzaparvovec

SRP-9003 (bidridistrogene fuzaparvovec) is an investigational gene therapy designed to treat limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease caused by mutations in the beta-sarcoglycan (SGCB) gene. The therapy utilizes a self-complementary adeno-associated virus serotype rh74 (rAAVrh74) vector to deliver a functional copy of the human SGCB gene to muscle cells. Expression of the transgene is controlled by the MHCK7 promoter, which is specifically engineered to drive robust protein production in skeletal and cardiac muscle. By restoring the beta-sarcoglycan protein, SRP-9003 aims to stabilize the dystrophin-associated protein complex, thereby protecting muscle fibers from damage and slowing disease progression. It was originally developed at the Center for Gene Therapy at Nationwide Children's Hospital and is currently being advanced in clinical trials by Sarepta Therapeutics.

Other names

AAVrh74.MHCK7.SGCBAAVrh-74.MHCK7.SGCBAAVrh 74.MHCK7.SGCB