Sarepta Therapeutics Inc. has announced that the U.S. Food & Drug Administration (FDA) has granted platform technology designation to the rAAVrh74 viral vector used in their investigational gene therapy SRP-9003. This therapy targets limb-girdle muscular dystrophy type 2E/R4. The recognition is one of the first of its kind, highlighting the reproducibility and adaptability of the technology across multiple therapeutic programs. The designation is part of the FDA's initiative to enhance the efficiency of science-driven processes, aiding in the streamlined development, manufacturing, and review of drug applications. This move allows Sarepta to leverage existing data from the platform to support future drug applications, underscoring its commitment to advancing treatments for rare genetic diseases.
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