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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.

Contents
Partners
Timeline
Funding rounds
Products
Acquisitions
SBA Awards
People
Further reading
Documentaries, videos and podcasts
Companies
News
References

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

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Acquisitions

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People

Name
Role
LinkedIn

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

Title
Author
Link
Type
Date

Sarepta Therapeutics enters manufacturing partnership for DMD treatment

Maggie Lynch

https://www.biopharma-reporter.com/Article/2018/10/17/Sarepta-Therapeutics-enters-manufacturing-partnership-for-DMD-treatment

Web

Sarepta Therapeutics shares soar 50% on positive preliminary results for muscular dystrophy gene therapy

Angelica LaVito

https://www.cnbc.com/2018/06/19/sarepta-therapeutics-shares-soar-on-preliminary-gene-therapy-data.html

News

Documentaries, videos and podcasts

Title
Date
Link

Companies

Company
CEO
Location
Products/Services

News

Title
Author
Date
Publisher
Description
Aducanumab Saga Continues as FDA Committee Members Resign Over Approval | BioSpace
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.
Gene Therapy Continues Edging into the Mainstream
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.
Sarepta Therapeutics' Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001
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
Sarepta thinks it may have a more effective DMD drug, but new side effects could be a problem
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.
MDA Celebrates FDA Approval of Amondys 45 for Treatment of DMD Amenable to Exon 45 Skipping
The Muscular Dystrophy Association
February 25, 2021
www.prnewswire.com:443
/PRNewswire/ -- The Muscular Dystrophy Association (MDA) today celebrated the decision by the US Food and Drug Administration (FDA) to grant accelerated...
SHAREHOLDER ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Sarepta Therapeutics, Inc. - SRPT
Pomerantz LLP
February 5, 2021
www.prnewswire.com:443
/PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Sarepta Therapeutics, Inc. ("Sarepta" or the "Company")(NASDAQ: SRPT). Such...
SRPT INVESTIGATION ALERT: Bernstein Liebhard is Investigating Sarepta Therapeutics, Inc. For Violations of the Securities Laws
Bernstein Liebhard LLP
January 8, 2021
www.prnewswire.com:443
/PRNewswire/ -- Bernstein Liebhard, a nationally acclaimed investor rights law firm, is investigating potential securities fraud claims on behalf of...
Sarepta's DMD gene therapy fails phase 2 motor function test, sinking stock
Nick Paul
January 8, 2021
FierceBiotech
Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy has failed to beat placebo in a phase 2 clinical trial. Functional motor ability scores in the SRP-9001 arm were statistically no better than in the placebo group, almost halving Sarepta's stock price and raising doubts about a key program.
Sarepta Therapeutics to Commence Dosing of the Next Study with Commercial Process Material for the SRP-9001 Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy
BioSpace
November 5, 2020
BioSpace
Dosing with SRP-9001 commercial-process material to proceed followingdiscussions with U.S. FDA
Sarepta bags drug to fully unlock DMD gene therapy opportunity
Nick Paul
July 2, 2020
FierceBiotech
Sarepta Therapeutics has secured a license to a drug that could enable more patients to receive its gene therapies. The deal grants Sarepta the exclusive right to use Hansa Biopharma's imlifidase to eliminate neutralizing antibodies in Duchenne muscular dystrophy and limb-girdle muscular dystrophy patients.
Sarepta's Gene Therapy for Limb-Girdle Muscular Dystrophy Shows Promise
Mark Terry
June 9, 2020
BioSpace
On Monday, Sarepta Therapeutics announced positive results from a small study of SRP-9003, its gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E).
Sarepta Therapeutics Announces Positive Expression and Functional Data From the SRP-9003 Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E
BioSpace
June 8, 2020
BioSpace
-- In post-treatment muscle biopsies, clinical trial participants in the high-dose cohort showed a dose-dependent increase in transduction and expression when compared with the low-dose cohort, with a mean of 72% beta-sarcoglycan (beta-SG) positive fibers, as measured by immunohistochemistry (IHC), substantially exceeding the pre-defined 50% measure for success -- -- A mean signal intensity of 73% in the high-dose group was observed compared to normal control -- -- A mean beta-sarcoglycan
Sarepta Therapeutics and Dyno Therapeutics Announce Agreement to Develop Next-Generation Gene Therapy Vectors for Muscle Diseases
BioSpace
May 11, 2020
BioSpace
Agreement leverages Sarepta's leadership in gene therapy for neuromuscular and cardiovascular diseases and Dyno's CapsidMap artificial intelligence platform to design AAV vectors
Dyno Explodes Out of Stealth Mode with $2 Billion in Deals with Biopharma Companies
Mark Terry
May 11, 2020
BioSpace
Dyno says that with its research-and-development and collaboration deals with biopharma companies, it is potentially eligible for more than $2 billion in upfront payments, research support, and various milestones and option fees.
Roche enters $1.15 billion licensing deal for Sarepta gene therapy
Reuters Editorial
December 23, 2019
U.S.
Roche entered into a $1.15 billion licensing agreement with Sarepta Therapeutics to obtain the right to launch and commercialize Sarepta's investigational gene therapy for Duchenne muscular dystrophy (DMD) outside the United States.

References

sarepta.com
Is a
Company

Company attributes

Industry
Biotechnology
Genetic engineering
Gene therapy
RNA
Healthcare
Pharmaceutical industry
Drug discovery
‌
Biopharmaceutical
...
Location
Cambridge, Massachusetts
Corvallis, Oregon
Andover, Massachusetts
United States
CEO
‌
Douglas S. Ingram
Founder
Marwan Fawaz
Crunchbase
crunchbase.com/organiz...apeutics
Date incorporated
1980
Full address
215 1ST ST STE 415 CAMBRIDGE, MA 02142 UNITED STATES
CIK number
873,303
Place of incorporation
Massachusetts
DUNS Number
121653406
IRS number
930,797,222
Founded date
1980
Total funding amount (USD)
430,499,960
Latest funding round date
February 2020
Competitors
Avidity Biosciences
Stock symbol
SRPT
Stock exchange
NASDAQ

Other attributes

Country
United States
Headquarters
Cambridge, Massachusetts
Invested in
Myonexus Therapeutics
StrideBio
AavantiBio
Market
Biotechnology
SIC code
2,834
Wikidata ID
Q15109846

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