Sarepta Therapeutics (NASDAQ:SRPT) -- formerly dba AVI BioPharma, and before that Antivirals Inc - is a biopharmaceutical company now outgrowth SBIR eligibility and focused on discovery and development of RNA-based therapeutics for the treatment of rare, infectious, and other diseases. Previously doing business in the Pacific Northwest - and now located in Cambridge MA - the firm's lead product candidate is Eteplirsen, an antisense phosphorodiamidate morpholino oligomer therapeutic. Now in Phase III clinical development stage, the product is used in treatment of individuals with Duchenne muscular dystrophy (DMD), a rare genetic muscle-wasting disease caused by the absence of dystrophin. The company also focuses on developing therapeutics for the treatment of drug-resistant bacteria, Becker muscular dystrophy, progeria, adult onset pompe, lupus and graft-versus-host diseases; and other rare, genetic, anti-infective, neuromuscular, and central nervous system diseases. The company is also involved in developing treatments that are in Phase I clinical trials for infectious diseases, including AVI-7288 for Marburg virus; and AVI-7100 for H1N1 influenza virus.
