Sarepta Therapeutics Receives FDA Platform Technology Designation for its rAAVrh74 Viral Vector

CAMBRIDGE, Mass.-June 4, 2025 -(BUSINESS WIRE)–Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, announced today that the U.S. Food & Drug Administration (FDA) has granted platform technology designation to its rAAVrh74 viral vector. This AAV vector is a key component of Sarepta’s investigational gene therapy, SRP-9003 (bidridistrogene xeboparvovec), which is being developed to treat limb-girdle muscular dystrophy type 2E/R4.

“This designation is a significant recognition by the FDA, acknowledging the reproducibility and adaptability of our rAAVrh74 AAV technology across multiple therapeutic programs,” said Dr. Louise Rodino-Klapac, Sarepta’s chief scientific officer and head of research & development. “It reinforces the consistency of data we’ve seen with this AAVrh74 in our clinical programs and highlights Sarepta’s ongoing commitment to accelerating transformative treatments for rare genetic diseases like LGMD type 2E/R4.”

What is Platform Technology Designation?

The FDA’s platform technology designation program is a crucial tool designed to streamline drug development, manufacturing, and review processes, particularly for rare disease therapies. It allows drug sponsors to leverage existing data from designated technologies when submitting applications for investigational new drugs (INDs), new drug applications (NDAs), or biologic license applications (BLAs).

To qualify, a technology must be well-understood and reproducible. This includes various elements like nucleic acid sequences, molecular structures, mechanisms of action, delivery methods, or AAV vectors, provided they can support the development of multiple drugs or biologics. A platform may also be considered if it’s already part of an approved drug and shows potential for use in other drugs without negative impacts on quality, manufacturing, or safety, including using the same manufacturing process. This designation helps to accelerate the development of innovative AAV gene therapies.

About SRP-9003 (bidridistrogene xeboparvovec)

SRP-9003 is an investigational gene therapy designed for systemic delivery to skeletal, diaphragm, and cardiac muscles. This AAV-based therapy aims to deliver a full-length beta-sarcoglycan transgene using the MHCK7 promoter. This promoter was specifically chosen for its ability to robustly express in the heart, which is vital for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4. Many individuals with this condition tragically succumb to pulmonary or cardiac complications, making this AAV gene therapy a crucial potential treatment.