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

The FDA has designated the viral vector rAAVrh74, which Sarepta Therapeutics, Inc. (NASDAQ:SRPT) uses in its gene therapy candidate SRP-9003 for limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4), as a platform technology.

A laboratory technician in a white coat holding a microscope and examining a vial of biopharmaceuticals.

Future approvals of treatments utilizing the same delivery technology might be accelerated by this designation. Phase III EMERGENE studies for SRP-9003, sometimes referred to as bidridistrogene xeboparvovec, are anticipated to yield data in the middle of 2025, and the FDA may file a proposal later this year.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) uses the viral vector rAAVrh74, which is produced from rhesus monkeys, in a number of neuromuscular gene therapy initiatives. Only 14% of DMD patients had pre-existing antibodies, which suggests that the vector has low frequencies of these antibodies, which could improve efficacy and lower safety hazards.

The FDA has acknowledged the vector’s constancy and adaptability, according to Sarepta Therapeutics, Inc. (NASDAQ:SRPT)’s CSO, Louise Rodino-Klapac. The firm can now expedite development throughout its gene therapy pipeline by reusing clinical data, which will cut the timeframe for future therapies of neuromuscular diseases.

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