Sarepta Therapeutics

    [td_text_with_title custom_title=”Company description”]Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. Our diverse pipeline includes eteplirsen, our lead program for Duchenne muscular dystrophy, as well as potential treatments for some of the world’s most lethal infectious diseases. We aim to build a leading, independent biopharmaceutical company dedicated to translating RNA-based science into transformational therapies for patients who face significant unmet medical needs.[/td_text_with_title]


    Eteplirsen, Sareptha´s lead investigational exon-skipping product candidate in clinical trials, is designed to skip exon 51 in the dystrophin gene. We also have other drug candidates in early stage preclinical development that are designed to skip exons 45, 53, 44, 52, 50, 55 and 8. We are committed to exploring the potential of our technology to [...]
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    Sarepta Therapeutics – knæk og bræk

    Sarepta Therapeutics har en spændende dag foran sig i dag. I fredags steg aktien 35%, men det sker langtfra på baggrund af en fundamental god udvikling i selskabet. Selskabets markedsværdi er nemlig styrtdykket 60% efter at FDA´s advisory panel i Januar udtrykte tvivl om effekten af deres eneste potentielle lægemiddel, Eterplisen, der retter sig mod Duchenne muscular dystrophy...

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    [/td_text_with_title][td_text_with_title custom_title=”From Wikipedia”]

    Sarepta Therapeutics Inc. (NASDAQSRPT) is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States. Incorporated in 1980, the company maintains some laboratory capability in Corvallis, Oregon. As of 2014[update], the company does not commercialize any approved drug.[1] As of 2008, Sarepta has 170 issued medical patents, and over 150 patents pending.[2] The company changed its name from AVI BioPharma and stock symbol from AVII in July 2012 to Sarepta Therapeutics and SRPT respectively.[3]


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

    In 2012 the company moved a second time, to Cambridge, Massachusetts. At the time, CEO Chris Garabedian indicated the move was motivated by the need to recruit expertise in rare diseases.[10] The Corvallis laboratory facility was closed in 2016.


    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 use in antisense therapy. In one application, translation blocking, a morpholino oligomer binds to messenger RNA produced by a known disease-causing gene to prevent it from being translated into protein. 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, Duchenne muscular dystrophy (DMD), and infectious diseases. Their greatest success thus far has been in DMD and as antiviral agents.

    The Morpholino drug eteplirsen, targeting exon 51 of the dystrophin mRNA, has been approved as a human therapeutic by the FDA in 2016[11] and clinical trials for Morpholinos targeting other exons are ongoing. 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 March 2013, the Company announced positive results from a non-human primate study of AVI-7288, the drug candidate for treatment of Marburg virus infection. The results showed that intramuscular administration of AVI-7288 resulted in survival rates up to 100 percent in monkeys exposed to this fatal virus. These results are similar to those in previous studies when the drug was given by intravenous injection.[12]

    In addition to development of Morpholinos as therapeutics, AVI has conducted six human trials for colorectal and pancreatic cancers using their cancer vaccine AVICINE.


    1. ^ "Here's why a small biotech like Sarepta has attracted so much attention". Boston Business Journal. 
    2. ^ Sarepta Therapeutics official website
    3. ^ "AVI BioPharma changes name, splits stock". Portland Business Journal. July 12, 2012. Retrieved 17 July 2012. 
    4. ^ Summerton, J (2005). "Morpholino Antisense Oligos: Applications in Biopharmaceutical Research". Innovations in Pharmaceutical Technology. No. Sept. Retrieved 29 Oct 2012.  | archiveurl =
    5. ^ a b Moody, Robin J. (November 5, 2003). "AVI BioPharma's losses shrinking". Portland Business Journal. Retrieved 2009-10-24. 
    6. ^ Moody, Robin J. (June 12, 2003). "AVI BioPharma stock reaches new 52-week high". Portland Business Journal. Retrieved 2009-10-24. 
    7. ^ a b c d DiMesio, Robbie (July 30, 2009). "AVI BioPharma moving HQ out of Oregon". The Oregonian. Retrieved 2009-10-24. 
    8. ^ "AVI BioPharma loses $19.7M in Q2". Portland Business Journal. August 10, 2009. Retrieved 2009-10-24. 
    9. ^ a b "AVI BioPharma gets $11.5M military contract". Portland Business Journal. October 5, 2009. Retrieved 2009-10-24. 
    10. ^ Timmerman, Luke. Sarepta Moves From Seattle to Boston for the Talent. 7 September 2012.
    11. ^
    12. ^ Sarepta's Marburg Drug Shows High Survival Rates After Intramuscular Delivery in Non-Human Primates. Marketwire, 4 March 2013.

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