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Last €23.59 EUR
Change Today +2.42 / 11.43%
Volume 2.1K
AB3A On Other Exchanges
As of 3:44 PM 05/22/15 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-based therapeutics for the treatment of rare and infectious diseases. The company is primarily focused on advancing the development of its disease-modifying Duchenne muscular dystrophy (DMD) drug candidates, including its priamry product candidate, eteplirsen. It is also focused on developing therapeutics for the treatment of infectious diseases, including its primary infectious disease program aimed at the development of a drug candidate for the Marburg hemorrhagic fever virus. DMD Program The company’s major program is designed to address specific gene mutations that result in DMD by forcing the genetic machinery to skip through an adjacent contiguous piece (one or more exons) of RNA, and thus, restore the ability of the cell to express a new, truncated but functional, dystrophin protein. Eteplirsen: Eteplirsen is an antisense PMO-based therapeutic in clinical development for the treatment of individuals with DMD who have an error in the gene coding for dystrophin that could be treated by skipping exon 51. Eteplirsen targets the most frequent series of mutations that cause DMD. Eteplirsen has been granted orphan drug designation in the United States and European Union. In addition to its major product candidate, eteplirsen, the company is pursuing development of additional exon-skipping drugs, to support its broad-based development program for the treatment of DMD. To support certain activities to enable an Investigational New Drug (IND) for an exon 45-skipping therapeutic, the company is collaborating with Children’s National Medical Center in Washington, D.C. and the Carolinas Medical Center (in Charlotte, N.C.). To support certain clinical proof of concept studies and IND-enabling activities for an exon 53-skipping therapeutic, the company is collaborating 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 United States. The company is developing exon 50 utilizing its own research and development capabilities. Infectious Disease Programs The company is implementing its RNA-based technology platforms in its infectious disease programs for the development of therapeutics to treat infectious diseases, such as Marburg and influenza. DoD has provided financial support for its development of therapeutics designed to treat Ebola, Marburg and influenza viruses. The company has also entered into an agreement with the National Institute of Allergy and Infectious Diseases, or NIAID, part of NIH, under which NIAID is providing clinical support for the development of its therapeutic candidate for the treatment of influenza. Hemorrhagic Fever Virus Programs The company’s infectious disease therapeutic programs use its translation suppression technology and apply its proprietary PMO-plus chemistry backbone, an advanced generation of its base PMO chemistry backbone that selectively introduces positive backbone charges to improve selective interaction between the drug and its target. Its translation suppressing technology is based on Translation Suppressing Oligomers, which are PMO-based compounds that stop or suppress the translation of a specific protein by binding to their specific target sequence in mRNA. The company is pursuing development and regulatory approval of its Marburg hemorrhagic fever virus product candidate under the FDA’s ‘Animal Rule’. Marburg Virus AVI-7288 is designed for post-exposure prophylaxis after documented or suspected exposure to Marburg virus. Marburg hemorrhagic fever is a severe and fatal disease in humans. For Marburg virus infection, its primary product candidate is AVI-7288. In 2013, with the support of DoD’s Joint Project Manager Medical Countermeasure Systems, in non-human primate study, the company completed an evaluation of the feasibility of in intramuscular route of administration using AVI-7288, including an evaluation of the tolerability, pharmacokinetics, and efficacy of intramuscular AVI-7288. In 2013, the com

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