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Sarepta Therapeutics, Inc.

Pipeline8 assets

  • SRP-1001SRP-1001 / FSHD1 / siRNA / Neuromuscular
    Facioscapulohumeral muscular dystrophy, Type 1 (FSHD1)
  • SRP-1003SRP-1003 / DM1 / siRNA / Neuromuscular
    Myotonic dystrophy, Type 1 (DM1)
  • SRP-1002SRP-1002 / siRNA / Pulmonary
    Idiopathic pulmonary fibrosis (IPF)
  • SRP-1004SRP-1004 / siRNA / Neurology
    Spinocerebellar ataxia type 2 (SCA2)
  • SRP-1005SRP-1005 / siRNA / Neurology
    Huntington's disease (HD)
  • SRP-1007SRP-1007 / siRNA / Neurology
    Spinocerebellar ataxia type 1 (SCA1)
  • SRP-1006SRP-1006 / siRNA / Neurology
    Spinocerebellar ataxia type 3 (SCA3)
  • bidridistrogene xeboparvovecSRP-9003 / LGMD2E/R4 β-sarcoglycan / Gene Therapy / Neuromuscular
    Limb-girdle Muscular Dystrophy

Latest news15 items

All news

Sarepta Announces FDA Acceptance of sNDAs for AMONDYS 45® and VYONDYS 53®

– Accepted for review with target action date of February 28, 2027 CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jun. 30, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted

Sarepta Therapeutics to Participate in Upcoming Investor Conferences

CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jun. 1, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will participate in the following investor conferences in June: Goldman Sachs 47 th Annual Global

Sarepta Therapeutics to Announce First Quarter 2026 Financial Results

CAMBRIDGE, Mass. --(BUSINESS WIRE)--Apr. 22, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report first quarter 2026 financial results after the Nasdaq Global Market closes on Wednesday, May 6, 2026. Subsequently, at 4:30 p.m.

Sarepta to Share First Clinical Data from siRNA Pipeline Targeting FSHD1 and DM1

CAMBRIDGE, Mass. --(BUSINESS WIRE)--Mar. 24, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Wed., March 25, 2026, at 8:30 am Eastern Time, the Company will host a webcast and conference call to present the early

Results Call • Phase 1/2

Sarepta Provides Regulatory Update on AMONDYS 45® and VYONDYS 53®

– Following feedback from FDA, Company intends to submit supplemental new drug applications to FDA by the end of April 2026 requesting conversion to traditional approval CAMBRIDGE, Mass. --(BUSINESS WIRE)--Mar. 19, 2026-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic

Sarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne

- Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the ENDEAVOR study - The enhanced immunosuppressive regimen is designed to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF) associated with AAV gene therapy in

Trial Start • Phase 1

Sarepta Therapeutics to Present New Long-Term and Safety Data Across Gene Therapy and Exon-Skipping Programs at 2026 Muscular Dystrophy Association Clinical & Scientific Congress

Several abstracts, including a late-breaking podium presentation and posters, bring forward accumulating long-term efficacy, safety and caregiver-reported insights that deepen understanding of dystrophin restoration and its impact in Duchenne CAMBRIDGE, Mass. --(BUSINESS WIRE)--Feb.

Presentation / Abstract • Phase 3

Sarepta Therapeutics Announces Commercial Launch of ELEVIDYS in Japan

- Japan now offers ELEVIDYS gene therapy to children with Duchenne muscular dystrophy aged 3 years to less than 8 years - Company is eligible to receive a $40 million milestone payment upon first commercial sale in Japan CAMBRIDGE, Mass. --(BUSINESS WIRE)--Feb. 24, 2026-- Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. • Endpoint Arena