Sarepta Therapeutics
NASDAQ:SRPTBiotechCambridge, Massachusetts, United States · Est. 1995 · 700 employees
Sarepta Therapeutics is a biotechnology company developing precision genetic medicine treatments for rare diseases, particularly muscular dystrophies.
4
Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
3
Indications↗
Distinct diseases across all programs
40
Clinical Trials↗
All sponsored trials (all statuses)
11
Orange Book Patents
Across 3 drugs with upcoming expirations
Pipeline
Pipeline Snapshot
By Phase
4 drugs in development
Approved2
Phase 33
Preclinical1
By Therapeutic Area
top 2
Rare Disease
480%
Neurology
120%
R&D Momentum
37/100
Pipeline Velocity
0/100
Trial Completion Rate
59/100
Pipeline Concentration
68/100
Pipeline Freshness
59/100
Strategic Signals
- ↗Mature portfolio may be looking to in-license early assets
- ↗Active in Neurology but thin pipeline — potential licensing opportunity
Activity
Key Events
Jun 25, 2026TrialAn Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)
Mar 12, 2026TrialESSENCE-53: Golodirsen in DMD Exon 53
Mar 10, 2026TrialMOMENTUM: Casimersen in DMD Exon 45
Mar 10, 2026TrialEMBARK: Delandistrogene Moxeparvovec Gene Therapy in DMD
Feb 28, 2026TrialESSENCE: Eteplirsen Long-Term Extension in DMD
Jun 30, 2025TrialA Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants
Jan 9, 2025TrialStudy 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
Feb 7, 2024TrialAn Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
4 more events
Jan 29, 2024TrialA Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
Jan 15, 2024TrialA Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
Feb 25, 2021FDAAmondys 45 — AMONDYS 45
Dec 12, 2019FDAVyondys 53 — VYONDYS 53
Details
Company Info
Company Overview
$5.5B
Mkt Cap
$1.2B
Revenue
—
Cash
CEODoug Ingram
Founded1995
Employees700
HQCambridge, Massachusetts, United States
ExchangeNASDAQ:SRPT
Websitesarepta.com
LinkedInCompany Page
Therapeutic Focus
Rare DiseaseNeurologyInfectious Disease
Pipeline
Drug Pipeline
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Neurology
Neurology
Muscular Dystrophy
SRP-9003Phase 3
Rare Disease
Regulatory
Regulatory Timeline
20161 event
FDASep 19
Exondys 51 — EXONDYS 51
Application NDA206488 (NDA)
20191 event
FDADec 12
Vyondys 53 — VYONDYS 53
Application NDA211970 (NDA)
20211 event
FDAFeb 25
Amondys 45 — AMONDYS 45
Application NDA213026 (NDA)
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)