Sarepta Viral Vector Gets FDA Platform Technology Designation

Sarepta Therapeutics Inc. (NASDAQ:SRPT) is one of the best mid cap growth stocks to invest in now. Earlier on June 4, Sarepta Therapeutics announced that the US FDA has granted Platform Technology Designation to the rAAVrh74 viral vector. The vector is utilized in SRP-9003 (bidridistrogene xeboparvovec), which is Sarepta’s investigational gene therapy for the treatment of limb-girdle muscular dystrophy type 2E/R4 (or LGMD2E/R4), also known as beta-sarcoglycanopathy.

The designation is the first of its kind awarded by the FDA and marks a recognition of the reproducibility and adaptability of this technology across multiple therapeutic programs. The Platform Technology Designation program, which was conceived in 2023 with draft guidance rolled out in May 2024, aims to streamline drug development, manufacturing, and review processes for drug product applications, especially for rare diseases, by allowing sponsors to use prior data from designated technologies.

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

The rAAVrh74 viral vector is a key component in several of Sarepta’s gene therapy programs, including Elevidys, an already FDA-approved gene therapy for Duchenne muscular dystrophy. It is also part of SRP-9003, which delivers a full-length beta-sarcoglycan transgene using the MHCK7 promoter, chosen for its expression in the heart, a critical factor for LGMD2E/R4 patients who often face pulmonary or cardiac complications. The designation allows Sarepta to use evidence previously gathered from studies involving the vector in future INDs, NDAs, or BLAs.

Sarepta Therapeutics Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company that discovers and develops RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases.

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Disclosure: None. This article is originally published at Insider Monkey.