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AAV - CNGB3
Gene therapy targeting the CNGB3 gene
Gene therapy targeting the CNGB3 gene Used for Leber congenital amaurosis, Retinitis pigmentosa.
At a glance
| Generic name | AAV - CNGB3 |
|---|---|
| Sponsor | MeiraGTx UK II Ltd |
| Drug class | Gene therapy |
| Target | CNGB3 gene |
| Modality | Biologic |
| Therapeutic area | Ophthalmology |
| Phase | Phase 1 |
Mechanism of action
AAV-mediated gene therapy aims to restore vision in patients with inherited retinal diseases by delivering a healthy copy of the CNGB3 gene to the retina.
Approved indications
- Leber congenital amaurosis
- Retinitis pigmentosa
Common side effects
Key clinical trials
- Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
- Gene Therapy for Achromatopsia (CNGB3) (PHASE1, PHASE2)
- Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial) (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:
- AAV - CNGB3 CI brief — competitive landscape report
- AAV - CNGB3 updates RSS · CI watch RSS
- MeiraGTx UK II Ltd portfolio CI