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SRP-4053
SRP-4053 is an antisense oligonucleotide that modulates dystrophin production by promoting exon 53 skipping in Duchenne muscular dystrophy patients with mutations amenable to this approach.
SRP-4053 is an antisense oligonucleotide that modulates dystrophin production by promoting exon 53 skipping in Duchenne muscular dystrophy patients with mutations amenable to this approach. Used for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 53 skipping.
At a glance
| Generic name | SRP-4053 |
|---|---|
| Also known as | Golodirsen, VYONDYS 53 |
| Sponsor | Sarepta Therapeutics, Inc. |
| Drug class | Antisense oligonucleotide |
| Target | Dystrophin pre-mRNA (exon 53) |
| Modality | Small molecule |
| Therapeutic area | Neuromuscular/Genetic |
| Phase | Phase 3 |
Mechanism of action
SRP-4053 binds to exon 53 of the dystrophin pre-mRNA and causes the splicing machinery to skip this exon during mRNA processing. This allows the production of a shorter but functional dystrophin protein (micro-dystrophin), which can partially restore muscle function in patients with specific DMD mutations. The drug is designed for patients whose mutations can be corrected by exon 53 skipping.
Approved indications
- Duchenne muscular dystrophy (DMD) with mutations amenable to exon 53 skipping
Common side effects
- Injection site reactions
- Immune-mediated toxicity
- Glomerulonephritis
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)
- Phase I/II Study of SRP-4053 in DMD Patients (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- SRP-4053 CI brief — competitive landscape report
- SRP-4053 updates RSS · CI watch RSS
- Sarepta Therapeutics, Inc. portfolio CI