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.
3
Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
3
Indications↗
Distinct diseases across all programs
46
Clinical Trials↗
All sponsored trials (all statuses)
11
Orange Book Patents
Across 3 drugs with upcoming expirations
Pipeline
Pipeline Snapshot
By Phase
3 drugs in development
Approved2
Phase 32
Preclinical1
By Therapeutic Area
top 2
Rare Disease
480%
Neurology
120%
R&D Momentum
49/100
Pipeline Velocity
11/100
Trial Completion Rate
59/100
Pipeline Concentration
68/100
Pipeline Freshness
66/100
Strategic Signals
- ↗Mature portfolio may be looking to in-license early assets
- ↗Active in Neurology but thin pipeline — potential licensing opportunity
Activity
Recent Activity
Last 14 days
Nov 3, 2026
Enrolling by invitation
A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.'s Exon-Skipping Therapies in Patients With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
Oct 24, 2026
Enrolling by invitation
A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
Earlier
Oct 20, 2026TrialA Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study to Evaluate the Effect of SRP-1005 in Subjects With Huntington's Disease (INSIGHTT)
Oct 7, 2026TrialPhase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)
Oct 6, 2026TrialA Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 (SRP-1003) in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years
Oct 6, 2026TrialA Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 (SRP-1001) in Adult Patients and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Sep 12, 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
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 DiseaseNeurology
Pipeline
Drug Pipeline
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Neurology
Neurology
Muscular Dystrophy
SRP-9003Phase 3
Rare Disease
Regulatory
Regulatory Timeline
20162 events
FDASep 19
Exondys 51 — EXONDYS 51
Application NDA206488 (NDA)
FDASep 19
Exondys 51 — EXONDYS 51
Application NDA206488
20171 event
FDAFeb 22
Exondys 51 — EXONDYS 51
Application NDA206488
20182 events
FDAOct 11
Exondys 51 — EXONDYS 51
Application NDA206488
FDAFeb 8
Exondys 51 — EXONDYS 51
Application NDA206488
20192 events
FDADec 12
Vyondys 53 — VYONDYS 53
Application NDA211970 (NDA)
FDADec 12
Vyondys 53 — VYONDYS 53
Application NDA211970
20201 event
FDAJul 7
Exondys 51 — EXONDYS 51
Application NDA206488
20213 events
FDAFeb 25
Amondys 45 — AMONDYS 45
Application NDA213026 (NDA)
FDAFeb 25
Amondys 45 — AMONDYS 45
Application NDA213026
FDAFeb 11
Vyondys 53 — VYONDYS 53
Application NDA211970
20223 events
FDAJan 18
Exondys 51 — EXONDYS 51
Application NDA206488
FDAJan 18
Exondys 51 — EXONDYS 51
Application NDA206488
FDAJan 18
Exondys 51 — EXONDYS 51
Application NDA206488
20231 event
FDAMar 23
Amondys 45 — AMONDYS 45
Application NDA213026
20244 events
FDADec 27
Exondys 51 — EXONDYS 51
Application NDA206488
FDAJul 11
Amondys 45 — AMONDYS 45
Application NDA213026
FDAJun 4
Vyondys 53 — VYONDYS 53
Application NDA211970
FDAJun 4
Vyondys 53 — VYONDYS 53
Application NDA211970
20261 event
FDAJan 6
Amondys 45 — AMONDYS 45
Application NDA213026
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)