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 experienced a significant decline in its stock price, dropping 48% following the death of a second teenage patient linked to its gene therapy, Elevidys. Despite this setback, some investors, referred to as "whales," are demonstrating strong confidence in the company through substantial investments.
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