From Yahoo Finance: 2025-06-05 17:39:00

The FDA has granted Sarepta platform technology designation for its rAAVrh74 viral vector, used in gene therapy candidate SRP-9003 for limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4). Future approvals of treatments using this technology may be accelerated, with Phase III EMERGENE studies expected to yield data in mid-2025.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) utilizes the viral vector rAAVrh74, derived from rhesus monkeys, in various neuromuscular gene therapy projects. Only 14% of DMD patients had pre-existing antibodies, indicating low frequencies of these antibodies, potentially improving efficacy and reducing safety risks. The FDA has recognized the vector’s stability and versatility, allowing Sarepta to expedite development across its gene therapy pipeline by reusing clinical data, reducing future therapy timelines for neuromuscular diseases.

Read more: The FDA granted Sarepta’s rAAVrh74 Viral Vector a Platform Technology Designation