Sarepta, FDA and gene therapy
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The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.
FDA places clinical hold on Sarepta's LGMD gene therapy trials after three deaths and revokes platform tech designation due to safety concerns.
The FDA will request Sarepta Therapeutics stop all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, following two deaths tied to the product.