SRP-6004 / Sarepta Therapeutics - LARVOL DELTA

SRP-6006: a novel myotropic dual vector system to treat limb-girdle muscular dystrophy type 2B/R2 (ASGCT 2026) - "Taken together, our results provide the preclinical basis for the development of SRP-6006 as a therapy for LGMD2B/R2. Conclusion Compared with clinical candidate SRP-6004, SRP-6006 demonstrates enhanced potency in Bla/J mice and may provide meaningful benefit to patients with LGMD2B/R2 at lower effective doses."

CNS Disorders • Gene Therapies • Muscular Dystrophy • DYSF

A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related) (clinicaltrials.gov) - P1 | N=2 | Terminated | Sponsor: Sarepta Therapeutics, Inc. | Trial completion date: Aug 2028 ➔ Jun 2025 | Active, not recruiting ➔ Terminated | Trial primary completion date: Aug 2028 ➔ Jun 2025; Study is being terminated due to a business decision.

Trial completion date • Trial primary completion date • Trial termination • Gene Therapies • Muscular Dystrophy

A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related) (clinicaltrials.gov) - P1 | N=2 | Active, not recruiting | Sponsor: Sarepta Therapeutics, Inc. | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Gene Therapies • Muscular Dystrophy

A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related) (clinicaltrials.gov) - P1 | N=2 | Enrolling by invitation | Sponsor: Sarepta Therapeutics, Inc.

New P1 trial • Gene Therapies • Muscular Dystrophy • DYSF