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Company Description

Contact Info

215 First Street

Suite 415

Cambridge, MA 02142

United States

Phone: 617-274-4000


Sarepta Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The company targets a range of diseases and disorders through distinct RNA-targeted mechanisms of action. It focuses on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates, including its primary DMD product candidate, eteplirsen, designed to skip exon 51. The company is also developing therapeutics using its technology for the treatment of drug resistant bacteria and infectious, rare and other human diseases. Development Programs DMD: The company’s primary program, with a pipeline of 10 product candidates, focuses on the development of disease-modifying therapeutic candidates for DMD, a rare genetic muscle-wasting disease caused by the absence of dystrophin, a protein necessary for muscle function. The company is in the process of conducting various studies with eteplirsen. Eteplirsen, the company’s primary DMD product candidate, is an antisense phosphorodiamidate morpholino oligomer (PMO) therapeutic in Phase III clinical development for the treatment of individuals with DMD who have an error in the gene coding for dystrophin that is amenable to skipping exon 51. In 2015, the company announced the Food and Drug Administration (FDA) filing of its new drug application (NDA) for eteplirsen for the treatment of DMD amenable to exon 51 skipping. The company is pursuing development of additional exon-skipping drugs, to support its broad-based development program for the treatment of DMD. Its additional nine product candidates target skipping of exons 8, 35, 43, 44, 45, 50, 52, 53, and 55 and are at various stages of development. Exon 53: To support certain clinical proof of concept studies and investigational new drug (IND) -enabling activities for an exon 53-skipping therapeutic, the company announced in 2012 that the company is collaborating in the SKIP-NMD Consortium with University College London’s scientist, Professor Francesco Muntoni, M.D., the Dubowitz Neuromuscular Centre, the Institute of Child Health and other scientists from the European Union and the U.S. Exon 45: In collaboration with Children’s National Medical Center (CNMC) in Washington, D.C. and the Carolinas Medical Center (CMC) in Charlotte, North Carolina, the company developed an exon 45-skipping product candidate. The company’s DMD program is part of its larger pan-exon strategy for the development of drug candidates to address the most prevalent exon deletions in the DMD population. Anti-virals: The company remains active partners with the National Institutes of Health (NIH) and the National Institute of Allergy and Infectious Diseases (NIAID) for continued development of its influenza product candidate. Discovery and Research Programs The company’s discovery and research programs include collaborations with various parties and focus on developing therapeutics in rare, genetic, anti-bacterial, neuromuscular and central nervous system diseases. It is exploring the application of its proprietary PMO platform technology in various diseases. Rare Diseases: The company is researching the application of its proprietary peptide-conjugated PMO (PPMO) technology to regulate progerin protein in progeria patients and in other diseases. Anti-Bacterials: The company’s anti-bacterial program is focused on drug-resistant bacteria identified by the Centers for Disease Control and Prevention (CDC) as urgent or serious threats to the U.S. healthcare system. Proprietary Manufacturing Techniques The company has developed proprietary manufacturing and scale-up techniques that allow synthesis and purification of its product candidates to support clinical development, as well as commercialization. The company has entered into certain manufacturing and supply arrangements with third parties. Strategy The key elements of the company’s strategy include advancing the development of eteplirsen and its other drug candidates for the treatment of DMD to realize the product opportun

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