Serepta Therepeutics, formerly AVI BioPharma, is a biotechnology company that is headquartered in Cambridge, Massachusetts, and was founded in 1980 by Marwan Fawaz. The company is developing gene therapies and medicines for the treatment of neuromuscular diseases such as Duchenne Muscular Dystrophy (DMD), Limb-Girdle Muscular Dystrophy (LGMD), and Charcot-Marie-Tooth (CMT). Serepta Therapeutics creates their gene therapies and medicines using multiple therapeutic approaches which include RNA, gene therapy, and gene editing.
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The company has worked with phosphorodiamidate morpholino oligomer chemistries in order to provide therapies for muscular dystrophy. The PMOs that the company works with are synthetic molecules that are modeled after the natural framework of RNA, and has the nucleic acid bases found in RNA.
They are able to hide specific exons, or skip their location if they are missing in order to provide a proper connection and create a functional dystrophin protein.
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Funding rounds
People
Amit Patel
Investor
Bo Cumbo
Senior Vice President & Chief Commercial Officer
Bruce Girton
Employee
Diane Berry
Vice President, Global Health Policy and Government Affairs
Douglas Ingram
President and CEO
Gary Charbonneau
Senior Vice President, Global Regulatory Affairs
Gilmore O'Neill
Senior Vice President, R&D and Chief Medical Officer
Ian Estepan
Chief of Staff, Vice President, Corporate Affairs
Jason Gatlin
Employee
Joan Nickerson
Vice President, Human Resources
Louise Rodino-Klapac
Vice President, Gene Therapy
Quinn Viens
Employee
Sagar Desai
Employee
Sandy Mahatme
Executive Vice President, CFO & CBO
Sarah Higgins
Employee
suchith nareddy
Employee
Ty Howton
Senior Vice president, General Counsel and Corporate Strategy
Further reading
Sarepta Therapeutics enters manufacturing partnership for DMD treatment
Maggie Lynch
Web
Sarepta Therapeutics shares soar 50% on positive preliminary results for muscular dystrophy gene therapy
Angelica LaVito
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