Sarepta Therapeutics
- Country
- 🇺🇸United States
- Ownership
- Private, Public
- Established
- 1980-01-01
- Employees
- 1.3K
- Market Cap
- $13.4B
- Website
- http://www.sarepta.com
- Introduction
Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company, which is engaged in the discovery and development of therapeutics for the treatment of rare diseases. The company was founded on July 22, 1980, and is headquartered in Cambridge, MA.
Clinical Trials
123
Trial Phases
3 Phases
Drug Approvals
4
Drug Approvals
Clinical Trials
Distribution across different clinical trial phases (117 trials with phase data)• Click on a phase to view related trials
A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants
- Conditions
- Muscular Dystrophies, Limb-Girdle
- Interventions
- Drug: Corticosteroid
- First Posted Date
- 2025-05-01
- Last Posted Date
- 2025-08-06
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 15
- Registration Number
- NCT06952686
- Locations
- 🇺🇸
Children's Hospital of the King's Daughters, Norfolk, Virginia, United States
Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States
- Conditions
- Limb Girdle Muscular DystrophyLimb Girdle Muscular Dystrophy Type 2D/R3
- Interventions
- First Posted Date
- 2024-12-24
- Last Posted Date
- 2025-04-27
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 4
- Registration Number
- NCT06747273
- Locations
- 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇺🇸Children's Hospital of the King's Daughters, Norfolk, Virginia, United States
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- First Posted Date
- 2024-09-23
- Last Posted Date
- 2024-09-23
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 300
- Registration Number
- NCT06606340
- Locations
- 🇺🇸
Arkansas Children's Hospital Research Institute, Little Rock, Arkansas, United States
🇺🇸Children's Hospital Los Angeles, Los Angeles, California, United States
🇺🇸University of California Davis Medical Center, Sacramento, California, United States
A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
- Conditions
- Duchenne Muscular Dystrophy
- First Posted Date
- 2024-09-19
- Last Posted Date
- 2025-07-14
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 10
- Registration Number
- NCT06597656
- Locations
- 🇺🇸
University of Florida, College of Medicine, Gainesville, Florida, United States
🇺🇸Washington University School of Medicine in St. Louis, Saint Louis, Missouri, United States
🇺🇸Nationwide Children's Hospital, Columbus, Ohio, United States
An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Genetic: Delandistrogene MoxeparvovecDrug: Standard of Care
- First Posted Date
- 2024-02-21
- Last Posted Date
- 2025-08-11
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 500
- Registration Number
- NCT06270719
- Locations
- 🇺🇸
All Children's Research Institute, Inc, Saint Petersburg, Florida, United States
🇺🇸OHSU Healthcare (Oregon Health and Science University), Portland, Oregon, United States
🇺🇸Arkansas Children's Hospital, Little Rock, Arkansas, United States
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News
Schrödinger Discontinues Blood Cancer Drug SGR-2921 After Two Patient Deaths in Phase I Trial
Schrödinger has terminated development of its CDC7 inhibitor SGR-2921 after two patient deaths in a Phase I trial for relapsed/refractory acute myeloid leukemia and high-risk myelodysplastic syndromes.
FDA Official Dr. Vinay Prasad Resigns After Three Months Amid Sarepta Gene Therapy Controversy
Dr. Vinay Prasad resigned from his FDA leadership position after less than three months following criticism from right-wing activists and pressure from the White House.
Arrowhead Pharmaceuticals Receives $100 Million Milestone Payment for ARO-DM1 Myotonic Dystrophy Treatment
Arrowhead Pharmaceuticals earned a $100 million milestone payment from Sarepta Therapeutics after reaching enrollment targets for ARO-DM1, an RNAi therapeutic for type 1 myotonic dystrophy.
FDA Appoints Biotech Veteran George Tidmarsh to Lead Drug Regulation Center Amid Agency Restructuring
The FDA has appointed Dr. George Tidmarsh, a former pharmaceutical executive and cancer specialist, to direct the Center for Drug Evaluation and Research, the agency's largest division responsible for reviewing drug safety and effectiveness.
Sarepta Therapeutics Implements $400 Million Cost-Cutting Plan Amid Regulatory Challenges for Gene Therapy
Sarepta Therapeutics announced a $400 million annual cost-savings initiative in July 2025, including a 36% workforce reduction of 500 employees to achieve financial sustainability through 2027.
CNS Oligonucleotide Licensing Deals Surge 339% to $2.81 Billion in 2024
Licensing agreements for CNS-targeted oligonucleotide therapeutics increased 339% from 2023 to 2024, reaching $2.81 billion in total deal value according to GlobalData's analysis.
FDA Awards Sarepta First Platform Technology Designation for Gene Therapy Vector
The FDA has granted Sarepta Therapeutics a platform technology designation for its rAAVrh74 viral vector, marking one of the first such awards since the program launched in 2024.
FDA Places Clinical Hold on Rocket Pharmaceuticals' Danon Disease Gene Therapy After Patient Death
The FDA has placed a clinical hold on Rocket Pharmaceuticals' Phase II gene therapy trial for RP-A501 after a patient developed capillary leak syndrome and subsequently died.
Satellos Reports Promising Efficacy Signals for Novel DMD Treatment in Adult Patients
Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area.
Sarepta Therapeutics Revises 2025 Guidance Following ELEVIDYS Safety Event and Operational Challenges
Sarepta Therapeutics reported strong Q1 2025 revenue of $744.9 million, beating analyst estimates by 7.4% with 80.2% year-over-year growth driven by increased ELEVIDYS usage.