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Last €33.51 EUR
Change Today -2.88 / -7.91%
Volume 0.0
As of 10:16 AM 10/6/15 All times are local (Market data is delayed by at least 15 minutes).
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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 is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD (Duchenne Muscular Dystrophy) drug candidates, including its lead 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 major program, with a pipeline of eight 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. Exon-Skipping Pipeline Eteplirsen: Eteplirsen, the company’s major DMD product candidate, is an antisense PMO (phosphorodiamidate morpholino oligomer) 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. Eteplirsen targets the most frequent series of mutations that cause DMD. Eteplirsen has been granted orphan drug designation in the U.S. and the European Union (E.U.). Additional DMD Product Candidates: In addition to the company’s major product candidate, eteplirsen, it is pursuing development of additional exon-skipping drugs, to support its broad-based development program for the treatment of DMD. The company’s additional seven product candidates target skipping of exons 8, 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 that it 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 E.U. and the U.S. In connection with this collaboration, the consortium received an E.U. Health Innovation-1 2012 collaborative research grant (grant agreement No. 305370) to support development of an exon 53-skipping therapeutic, based on the company’s PMO chemistry. In addition to this E.U. study, the company plans to further explore the safety and efficacy of SRP-4053 as part of an eteplirsen confirmatory study, which it plans to start in 2015. Exon 45: In collaboration with Children’s National Medical Center (CNMC) in Washington, D.C. and the Carolinas Medical Center (CMC) in Charlotte, N.C., the company has developed an exon 45-skipping product candidate. This collaboration is funded primarily through two grants, one from the Department of Defense’s Congressionally Directed Medical Research Program to CNMC and the other from the National Institute of Neurological Disorders and Stroke to the CMC. The collaboration has supported a series of Good Laboratory Practice toxicology studies for an exon 45-skipping drug candidate based on the company’s PMO chemistry. SRP-4045, designed to skip exon 45, is expected to advance to the clinical stage of development in 2015 as part of the eteplirsen confirmatory trial that the company plans to start in 2015. Exons 8, 44, 50, 52, and 55: Selection of lead sequences for product candidates designed to skip each of these exons are underway and the company plans to continue its pre-clinical development of these product candidates in 2015. Development Program: Infectious Diseases The company’s infectious disease therapeutic programs use its translation suppression technology and apply its proprietary PMOplus chemistry platform, an advanced generation of the company’s base PMO chemistry platform that selectively introduces positive backbone charges to improve selective interaction between the drug and its target. With the prior financial support of the U.S. gover


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