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Elevidys Helps Sarepta To First US FDA Platform Technology Designation

 
• By Bridget Silverman

Sarepta's rAAVrh74 vector, used in the marketed Duchenne muscular dystrophy gene therapy Elevidys and across the company's limb girdle muscular dystrophy pipeline, earned a platform designation as the lead LGMD candidate prepares for BLA filing.

The FDA's platform technology designation for Sarepta's rAAVrh74 vector could boost its pipeline of gene therapies for limb-girdle muscular dystrophies. (Shutterstock)
Key Takeaways
  • Sarepta's rAAVrh74 viral vector earned the first platform technology designation from the US FDA based on the likelihood of "significant efficiencies" in review, development or manufacturing and preliminary evidence for use by more than one therapy.

Sarepta Therapeutics is hoping to streamline development across its pipeline of gene therapies for limb-girdle...

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