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: Business Wire
-- Trial to assess efficacy on neurodevelopmental status of MPS IIIA patients -- CAMBRIDGE, Mass. & PARIS--(BUSINESS WIRE)-- Regulatory News: 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
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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