Sarepta’s rAAVrh74 Viral Vector Receives FDA Platform Technology Designation for Gene Therapy

On June 4, 2025, Sarepta Therapeutics announced that the FDA granted platform technology designation to its rAAVrh74 viral vector, which is used in the investigational gene therapy SRP-9003 for limb-girdle muscular dystrophy R4 (type 2E) 1 2 3 4 5.

This is among the first gene therapy programs to receive platform technology designation from the FDA, recognizing the vector's reproducibility, adaptability, and consistent data across Sarepta’s clinical programs 2.

The designation allows Sarepta to leverage prior data on the rAAVrh74 vector to streamline future Investigational New Drug (IND), New Drug Application (NDA), and Biologics License Application (BLA) filings, potentially accelerating development, manufacturing, and review processes for other gene therapies using the same vector 1 2 5.

SRP-9003 (bidridistrogene xeboparvovec) delivers a full-length beta-sarcoglycan transgene to skeletal, diaphragm, and cardiac muscle, aiming to treat patients with neuromuscular diseases who face risks of severe cardiac or pulmonary complications 1 2.

The FDA platform technology program is designed to improve efficiency in rare disease drug development by permitting sponsors to use existing data on a qualified platform to support new therapies, as long as quality, safety, and manufacturing standards are maintained 1 2 5.