Company Description:Sarepta Therapeutics, Inc. focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. The company is also developing AMONDYS 45, a product candidate that uses phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to skip exon 45 of the dystrophin gene. It has collaboration agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Lysogene; Duke University; Genethon; and StrideBio.