Sarepta Therapeutics, based in Cambridge, Massachusetts, focuses on RNA-targeted therapeutics for rare diseases, particularly Duchenne muscular dystrophy, and has developed four approved products. The company employs 1,314 people and has about 40 programs in its pipeline.
SRPT has been in the news recently: Sarepta Therapeutics, Inc. (SRPT) experienced a significant drop in its stock price after a third patient death linked to its gene therapy SRP-9004 raised investor concerns. Prior to this incident, the stock had risen following positive updates on the label expansion for its gene therapy Elevidys for Duchenne muscular dystrophy.
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