Sarepta Therapeutics, Inc.

We are a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, siRNA knockdown therapies, gene therapy, and other genetic therapeutic modalities for the treatment of rare diseases. We apply our proprietary, differentiated, and innovative technologies, and collaborate with strategic partners. We have developed multiple approved products for the treatment of Duchenne muscular dystrophy ("Duchenne") and are developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne and LGMD. We are also developing potential therapeutic candidates through our partnered program with Arrowhead for the treatment of Facioscapulohumeral muscular dystrophy ("FSHD"), myotonic dystrophy type 1 ("DM1"), Spinocerebellar ataxia ("SCA"), Idiopathic Pulmonary Fibrosis ("IPF"), Huntington's disease, and other neuromuscular and skeletal diseases. To date, we have developed and commercialized four approved products for the treatment of Duchenne: EXONDYS 51 (eteplirsen) Injection ("EXONDYS 51"), VYONDYS 53 (golodirsen) Injection ("VYONDYS 53"), AMONDYS 45 (casimersen) Injection ("AMONDYS 45"), and ELEVIDYS. We believe our proprietary technology platforms and collaborations can be used to develop novel pharmaceutical products to treat a broad range of diseases and address key currently-unmet medical needs.