Sarepta Therapeutics
USINFO | 2013-08-05 11:19
 
 
Sarepta Therapeutics Inc. (NASDAQ: SRPT) is a medical research and drug development company with corporate offices and research facility in Cambridge, Massachusetts, United States. Incorporated in 1980, the company maintains some laboratory capability in Bothell, Washington and Corvallis, Oregon. As of 2008, the company has 170 issued medical patents, and over 150 patents pending.[1] The company changed its name from AVI BioPharma and stock symbol from AVII in July 2012 to Sarepta Therapeutics and SRPT respectively.[2]

History
At its founding in 1980, the company was named AntiVirals, Inc.[3] After occupying several research laboratory spaces in Corvallis, the renamed AVI BioPharma Inc. opened a production laboratory in Corvallis, Oregon, in February 2002.[4] The company made headlines in 2003 when it announced work on treatments for SARS and the West Nile Virus.[4][5] In July 2009, the company announced they would move their headquarters from Portland, Oregon, north to Bothell, Washington, nearSeattle.[6] At that time the company led by president and CEO Leslie Hudson had 83 employees and quarterly revenues of $3.2 million.[6] AVI had yet to turn a profit nor developed any commercial products as of July 2009.[6] The company lost $19.7 million in the second quarter of 2009,[7] and then won a $11.5 million contract with the U.S. Department of Defense's Defense Threat Reduction Agency in October 2009.[8] The company had completed its move to Bothell by this time, but retained their Corvallis facility.[6][8] In 2012 the company moved its headquarters to Cambridge, MA.[9]

Products 
Its primary products are Morpholino oligomers (PMOs), synthetic nucleic acid analogs that were conceived of by James Summerton and invented by Summerton with Dwight Weller, and are being developed under the name NeuGene Antisense. Since Morpholino oligomers can form sequence-specific double-stranded complexes with RNA they are suitable for use in antisense therapy. In this application a Morpholino oligomer binds to messenger RNA produced by a known disease-causing gene to prevent it from being translated into protein. Alternatively, the Morpholinooligo can be targeted to pre-mRNA to alter splicing, a mechanism that shows promise to ameliorate some genetic diseases; Sarepta'sMorpholinodrug Eteplirsen is currently in clinical trial for skipping exon 51 of human dystrophin for treatment of some mutations causing Duchenne muscular dystrophy (DMD).
Morpholinos have been tested for a wide range of applications including prevention of cardiac restenosis after angioplasty, treatment of coronary artery bypass grafts, treatment of polycystic kidney disease, redirection of drug metabolism, and infectious diseases. Their greatest success thus far has been in DMD and as antiviral agents. Morpholinos have been used in preclinical studies to inhibit replication of a broad range of viruses, including influenza, West Nile Virus,SARS, Hepatitis C, dengue fever, Ebola and Calicivirus, all of which are single stranded RNA viruses. They are in advanced development for prevention and treatment of Ebola and Marburg viruses.
In addition to development of Morpholinos as therapeutics, Sarepta has conducted six human trials for colorectal and pancreatic cancers using their cancer vaccine AVICINE.
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