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Sarepta Announces Clinical Hold Lifted for its Duchenne Muscular Dystrophy Micro-dystrophin Gene Therapy Program
Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced today that the Food and Drug Administration (FDA) has lifted the clinical hold for the Company’s Duchenne muscular dystrophy (DMD) micro-dystrophin gene therapy program. Sarepta previously announced on July 25, 2018, that the FDA placed the program on clinical hold due to the presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid in a manufacturing lot. In response, and in collaboration with Nationwide Children’s Hospital, an action plan was developed and submitted to the FDA, including an audit of the plasmid supplier and a commitment to use GMP-s plasmid for all future production lots.
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