SRP-6004
Myotonic Dystrophy Type 1 (DM1)
Phase 1/2Active (siRNA); First clinical data 2026
Key Facts
Indication
Myotonic Dystrophy Type 1 (DM1)
Phase
Phase 1/2
Status
Active (siRNA); First clinical data 2026
Company
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biotech leader with an urgent mission to engineer and deliver precision genetic medicines for rare diseases. The company has achieved significant commercial success with multiple FDA-approved RNA-based exon-skipping therapies for Duchenne muscular dystrophy (DMD) and the landmark approval of ELEVIDYS, a gene therapy for DMD. Its strategy leverages a deep, multi-modal pipeline, a hybrid manufacturing model for scale, and a global commercial footprint to solidify its position as a dominant force in rare disease therapeutics.
View full company profileTherapeutic Areas
Other Myotonic Dystrophy Type 1 (DM1) Drugs
| Drug | Company | Phase |
|---|---|---|
| ATX-01 (ArthemiR trial) | ARTHEx Biotech | Phase 1/2 |
| AOC 1001 | Avidity Biosciences | Phase 1/2 |
| DYNE-101 | Dyne Therapeutics | Phase 1/2 |
| Undisclosed | Design Therapeutics | Preclinical |
| PGN-EDODM1 | PepGen | Phase 2 |