Sarepta announces pipeline progress for limb-girdle muscular dystrophy subtypes

Sarepta (SRPT) shared updates from its clinical programs focused on limb-girdle muscular dystrophy subtypes 2C/R5, 2D/R3, and 2E/R4. Following input from the FDA, Office of Therapeutic Products, Sarepta is cleared to proceed with dosing in Study SRP-9005-101 in the U.S. COMPASS is a first-in-human clinical study of SRP-9005, an investigational gene therapy for LGMD type 2C/R5, or gamma-sarcoglycanopathy. Enrollment and dosing is complete in Study SRP-9004-102. DISCOVERY is a phase 1, proof-of-concept study evaluating safety and expression of the alpha-sarcoglycan protein after treatment with SRP-9004, an investigational gene therapy for the treatment of LGMD type 2D/R3, or alpha-sarcoglycanopathy. Enrollment and dosing is complete in Study SRP-9003-301. EMERGENE is a phase 3 clinical trial of SRP-9003 for the treatment of LGMD type 2E/R4, or beta-sarcoglycanopathy. EMERGENE is a global study and the primary endpoint is the biomarker expression of beta-sarcoglycan protein. A pre-biologics license application meeting has occurred and the OTP has confirmed eligibility for the accelerated approval pathway for the program. Sarepta remains on track to submit a BLA to the FDA in the second half of 2025.