Sarepta Therapeutics Inc.

SRPT

Held by 13 specialist biotech funds

High Convergence

13 funds opened new positions. Next phase3 readout (SRP-9003): Mar 2025. Short interest: 25.9% of float.

BiotechnologyCEO: Michael E. SeverinoIPO: 1997Website ↗

Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company dedicated to the discovery and advancement of genetic treatment modalities, including RNA-targeted therapeutics and gene therapies, specifically for rare diseases. The company currently offers two approved treatments: EXONDYS 51, prescribed for Duchenne muscular dystrophy (DMD) patients with confirmed dystrophin gene mutations amenable to exon 51 skipping, and VYONDYS 53, for DMD patients with exon 53 skipping mutations. Sarepta's development pipeline includes AMONDYS 45, an exon-skipping product candidate utilizing phosphorodiamidate morpholino oligomer chemistry for exon 45; SRP-5051, a peptide-conjugated PMO designed to target exon 51 of dystrophin pre-mRNA; and gene therapy programs such as SRP-9001 for DMD and SRP-9003 for limb-girdle muscular dystrophies. The company maintains collaborative agreements with organizations like F. Hoffman-La Roche Ltd, Nationwide Children's Hospital, Lysogene, Duke University, Genethon, and StrideBio. Founded in 1980, Sarepta Therapeutics is headquartered in Cambridge, Massachusetts.

Catalysts

Phase 2NegativeQ4 2006AVI-4065 InjectionHepatitis CNCT00229749

Primary: Safety and tolerabilityEnrollment:Target 80NON_RANDOMIZED, TREATMENT

Phase 2PositiveQ1 2012AVI-4658 (Eteplirsen)Duchenne Muscular DystrophyNCT01396239

Phase 2PositiveQ4 2018eteplirsenDuchenne Muscular Dystrophy (DMD)NCT02420379

Phase 2Q1 2019Limb-Girdle Muscular Dystrophy, Type 2DNCT01976091

Phase 2Q1 2019Duchenne Muscular DystrophyNCT02310906

Phase 3ApprovedQ2 2019eteplirsenDuchenne Muscular Dystrophy (DMD)NCT02255552

Phase 2PositiveQ1 2021EteplirsenDuchenne Muscular DystrophyNCT03218995

Phase 2NegativeQ3 2021SRP-5051Muscular Dystrophy, DuchenneNCT03675126

Phase 2PositiveQ2 2023Duchenne Muscular DystrophyNCT03375164

Phase 3NegativeQ3 2023CasimersenDuchenne Muscular DystrophyNCT03532542

Phase 2NegativeQ4 2023VesleteplirsenDuchenne Muscular DystrophyNCT04004065

Phase 2NegativeQ1 2025Limb-Girdle Muscular Dystrophy, Type 2ENCT03652259

Phase 3Q1 2025SRP-9003Limb-girdle Muscular DystrophyNCT06246513

Primary: Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers (Baseline, Day 60)Enrollment:17/17Sites: 10NA, TREATMENT

Phase 3Q4 2026EteplirsenMuscular Dystrophy, DuchenneNCT03992430

Primary: Part 1: Incidence of Adverse Events (AEs) (Up to Week 148)Enrollment:160/160Sites: 59RANDOMIZED, QUADRUPLE, TREATMENT

Phase 2Q4 2026SRP-1003 IV InfusionMyotonic Dystrophy 1NCT06138743

Primary: Number of Participants with Treatment-emergent Adverse Events Over Time Through End of Study (EOS) (Single-dose phase (Part 1): Up to Day 90 (EOS); multiple-dose phase (Part 2): Up to Day 180 (EOS))Enrollment:Target 78Sites: 35RANDOMIZED, QUADRUPLE, TREATMENT

Phase 3Q2 2027Duchenne Muscular DystrophyNCT05881408

Primary: Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72 (Baseline, Week 72)Enrollment:Target 148Sites: 46RANDOMIZED, QUADRUPLE, TREATMENT

Phase 2Q4 2028SRP-1001 for InjectionMuscular Dystrophy, FacioscapulohumeralNCT06131983

Phase 3Q4 2030SRP-9005Muscular Dystrophies, Limb-GirdleNCT06952686

Phase 3Q4 2033Duchenne Muscular DystrophyNCT05967351

Phase 2TBDRESTEN-MPCoronary Artery Disease, Coronary RestenosisNCT00248066

Primary: Evaluate the potential efficacy of RESTEN-MP given intravenously upon confirmation of stent implantation and again at 24 hours post-stent implantationEnrollment:Target 50Sites: 3NON_RANDOMIZED, TREATMENT

Sarepta Therapeutics Inc.

Signal Score

Conviction + Catalyst

Fund Holdings

Position Trend

Cash Runway

Short Interest

Catalysts

Activist Filings

Conferences

Recent News & Research

Position Details

Know which insider moves are real

Also Held by These Funds

Research Links

Related Research