bidridistrogene xeboparvovec

Bidridistrogene xeboparvovec (SRP-9003) is an investigational gene therapy developed by Sarepta Therapeutics for the treatment of limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as R4 or beta-sarcoglycanopathy. The therapy utilizes the AAVrh74 viral vector to deliver a full-length, functional copy of the beta-sarcoglycan (SGCB) transgene to skeletal and cardiac muscle tissues. It employs the MHCK7 promoter, which is specifically designed to drive robust protein expression in the heart and skeletal muscles, addressing the primary sites of disease pathology. By restoring the production of the beta-sarcoglycan protein, which is absent in patients with LGMD2E, the therapy aims to stabilize the dystrophin-associated glycoprotein complex and prevent the progressive muscle wasting and cardiac complications associated with the disorder.

Other names

AAVrh74.MHCK7.SGCBAAVrh-74.MHCK7.SGCBAAVrh 74.MHCK7.SGCBbeta-sarcoglycan gene therapy