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SRP-4045
SRP-4045 is a micro-dystrophin gene therapy that aims to treat Duchenne muscular dystrophy by providing a functional copy of the dystrophin gene.
SRP-4045 is a micro-dystrophin gene therapy that aims to treat Duchenne muscular dystrophy by providing a functional copy of the dystrophin gene. Used for Duchenne muscular dystrophy.
At a glance
| Generic name | SRP-4045 |
|---|---|
| Also known as | Casimersen, AMONDYS 45 |
| Sponsor | Sarepta Therapeutics, Inc. |
| Drug class | Gene therapy |
| Target | Dystrophin gene |
| Modality | Small molecule |
| Therapeutic area | Muscular dystrophy |
| Phase | Phase 3 |
Mechanism of action
SRP-4045 uses a viral vector to deliver a micro-dystrophin gene to muscle cells, allowing them to produce a truncated but functional form of the dystrophin protein. This can help to alleviate the symptoms of Duchenne muscular dystrophy by reducing muscle damage and improving muscle function.
Approved indications
- Duchenne muscular dystrophy
Common side effects
- Muscle pain
- Fatigue
- Headache
Key clinical trials
- Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) (PHASE3)
- A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
- An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy (PHASE3)
- A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. (PHASE2)
- Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients (PHASE1)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |