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SRP-9003
SRP-9003 is an antisense oligonucleotide that reduces production of DMPK protein by targeting mutant DMPK mRNA in myotonic dystrophy type 1.
SRP-9003 is an antisense oligonucleotide that reduces production of DMPK protein by targeting mutant DMPK mRNA in myotonic dystrophy type 1. Used for Myotonic dystrophy type 1 (DM1).
At a glance
| Generic name | SRP-9003 |
|---|---|
| Also known as | scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec |
| Sponsor | Sarepta Therapeutics, Inc. |
| Drug class | Antisense oligonucleotide |
| Target | DMPK mRNA |
| Modality | Biologic |
| Therapeutic area | Neuromuscular/Genetic Disorders |
| Phase | Phase 3 |
Mechanism of action
SRP-9003 uses antisense technology to bind to and degrade mutant DMPK mRNA transcripts, thereby reducing the production of toxic DMPK protein that accumulates in muscle cells and causes progressive muscle weakness and dysfunction. By lowering DMPK protein levels, the drug aims to slow or halt disease progression in myotonic dystrophy type 1 (DM1), a genetic neuromuscular disorder.
Approved indications
- Myotonic dystrophy type 1 (DM1)
Common side effects
- Injection site reactions
- Flu-like symptoms
- Transient liver enzyme elevation
Key clinical trials
- A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (PHASE3)
- A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency) (PHASE1)
- Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency) (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-9003 CI brief — competitive landscape report
- SRP-9003 updates RSS · CI watch RSS
- Sarepta Therapeutics, Inc. portfolio CI