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CX501
CX501 is a gene therapy that aims to increase the production of dystrophin in patients with Duchenne muscular dystrophy.
CX501 is a gene therapy that aims to increase the production of dystrophin in patients with Duchenne muscular dystrophy. Used for Duchenne muscular dystrophy.
At a glance
| Generic name | CX501 |
|---|---|
| Sponsor | Tigenix S.A.U. |
| Target | dystrophin gene |
| Modality | Small molecule |
| Therapeutic area | Muscular Dystrophy |
| Phase | Phase 2 |
Mechanism of action
CX501 uses a viral vector to deliver a healthy copy of the dystrophin gene to muscle cells, allowing them to produce dystrophin protein and potentially reversing or halting disease progression.
Approved indications
- Duchenne muscular dystrophy
Common side effects
- Muscle weakness
- Muscle pain
- Fever
Key clinical trials
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:
- CX501 CI brief — competitive landscape report
- CX501 updates RSS · CI watch RSS
- Tigenix S.A.U. portfolio CI