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Sarepta Therapeutics

Sarepta Therapeutics

Sarepta Therapeutics is a biopharmaceutical company founded by Marwan Fawaz in 1980 and headquartered in Cambridge, Massachusetts developing precision genetic medicines for the treatment of rare neuromuscular diseases.

What is Sarepta Therapeutics?

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.

Partners

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.

Timeline

1980
Sarepta Therapeutics was founded by Marwan Fawaz.

Funding rounds

Acquisitions

Patents

Further Resources

Title
Author
Link
Type
Date

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

News

News

Title
Author
Date
Publisher
Description
By MATTHEW PERRONE
July 1, 2021
AP NEWS
WASHINGTON (AP) -- When a controversial Alzheimer's drug won U.S. approval, surprise over the decision quickly turned to shock at how long it might take to find out if it really works -- nine years.Drugmaker Biogen has until 2030 to complete a study confirming whether its new drug Aduhelm truly slows the brain-destroying disease.
Mark Terry
June 10, 2021
BioSpace
David S. Knopman and Joel S. Perlmutter both resigned from the committee after the approval of the drug aducanumab.
Mark Terry
May 25, 2021
BioSpace
Gene therapy is still very much cutting-edge with the potential to cure incurable genetic diseases. Here's some recent news in the space.
BioSpace
May 18, 2021
BioSpace
Results from the first 11 participants enrolled in Study 9001-103 ENDEAVOR showed robust transduction, delivering mean vector genome copies of 3.87 per nucleus Treated patients achieved mean micro-dystrophin expression levels of 55.4% of normal as measured by western blot
Annalee Armstrong
May 3, 2021
FierceBiotech
Sarepta Therapeutics says its RNA therapy for Duchenne Muscular Dystrophy, SRP-5051, could be more effective than the company's existing therapies, but a dose escalation study also dug up some new--and serious--adverse events in patients.
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References

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