Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfil...
Sarepta Therapeutics, Inc. (SRPT)
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Source: GlobeNewswire
-- Trial to assess efficacy on neurodevelopmental status of MPS IIIA patients -- CAMBRIDGE, Mass. and PARIS, Feb. 14, 2019 (GLOBE NEWSWIRE) -- Lysogene (FR0013233475 – LYS), a pioneering biopharmaceutical company specializing in gene therapy targeting central nervous system (CNS) diseases, and Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that the first patient has been dosed in AAVance, a global Phase 2-3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA). AAVance is a single-arm trial aimed at evaluating the effectiveness of a one-time delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase (SGSH) gene. MPS IIIA is caused by mutations in the SGSH gene, which is involved in producing an enzyme necessary for the breakdown and disposal of long chain sugar molecules. LAF-SAF302 is intended to deliver a
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News
- Earnings Beat: Sarepta Therapeutics, Inc. Just Beat Analyst Forecasts, And Analysts Have Been Updating Their Models [Yahoo! Finance]Yahoo! Finance
- Sarepta Therapeutics, Inc. (NASDAQ: SRPT) had its price target lowered by analysts at JPMorgan Chase & Co. from $177.00 to $175.00. They now have an "overweight" rating on the stock.MarketBeat
- Gene Therapy Pioneer Jerry R. Mendell, M.D., Named to Inaugural TIME100 Health List of the 100 Most Influential People in Global Health [Yahoo! Finance]Yahoo! Finance
- Gene Therapy Pioneer Jerry R. Mendell, M.D., Named to Inaugural TIME100 Health List of the 100 Most Influential People in Global HealthBusiness Wire
- Sarepta Elevidys accelerated approval criticized by ICER exec [Seeking Alpha]Seeking Alpha
SRPT
Earnings
- 5/1/24 - Beat
SRPT
Sec Filings
- 5/6/24 - Form 4
- 5/6/24 - Form 3
- 5/1/24 - Form 10-Q
- SRPT's page on the SEC website