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
- 2025-09-04
- 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
- 2025-09-10
- 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
- 2025-09-04
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 3
- Registration Number
- NCT06597656
- Locations
- 🇺🇸
University of Florida, College of Medicine, Gainesville, Florida, United States
🇺🇸Washington University School of Medicine in St. Louis, St 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
Sarepta Therapeutics Refinances $700M Convertible Notes to Strengthen Financial Position
Sarepta Therapeutics exchanged approximately $700 million of 1.25% convertible notes due 2027 for $602 million in new 4.875% notes due 2030, extending debt maturity by three years.
Kriya Therapeutics Raises $313M to Advance Gene Therapy Pipeline for Chronic Diseases
Kriya Therapeutics has secured $313 million in new funding to advance its pipeline of single-dose gene therapies targeting chronic conditions including geographic atrophy and trigeminal neuralgia.
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.
AAV Gene Therapy Pipeline Expands with 180+ Companies Developing 250+ Treatments Despite Recent Setbacks
Over 180 companies are actively developing more than 250 AAV-based gene therapies, representing a robust pipeline for treating genetic disorders including hemophilia, spinal muscular atrophy, and inherited retinal diseases.
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.