Sarepta Therapeutics (SRPT) creates RNA-based drugs for the treatment of serious and life threatening diseases. Its most advanced candidate is an intravenously delivered treatment for Duchenne Muscular Dystrophy [DMD]. In addition to the promising DMD program, the company also has drugs for the treatment of ebola, Marburg and influenza viruses in clinical Phase 1 trials.
RNA drugs without the R
The company uses a phosphorodiamidate morpholino oligomer [PMO] antisense platform that allows for a diverse range of therapeutic candidates. The technology is far simpler than its name implies. Natural RNA is a string of nucleic acid bases (adenine, cytosine, guanine, and thymine) held together by backbone of recurring ribose rings. Sarepta's PMOs use the same nucleic acid bases,...
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