Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AAV- CNGB3 · 1 trial · 1 indication
The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences * Severe unresponsive inflammation * Infective endophthalmitis * Ocular malignancy * Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)
| Arm | Type | Description |
|---|---|---|
| Biological-Low dose AAV - CNGB3 | EXPERIMENTAL | Subretinal administration of a single low dose of AAV - CNGB3 |
| Biological-Medium dose AAV - CNGB3 | EXPERIMENTAL | Subretinal administration of a single intermediate dose of AAV - CNGB3 |
| Biological-High dose AAV - CNGB3 | EXPERIMENTAL | Subretinal administration of a single high dose of AAV - CNGB3 |
| Name | Type | Description |
|---|---|---|
| AAV - CNGB3 | BIOLOGICAL | Comparison of different dosages of AAV-CNGB3 |
Inclusion Criteria: * Are aged 3 years or older * Have achromatopsia confirmed by a retinal specialist (CI or PI) Exclusion Criteria: * Are females who are pregnant or breastfeeding * Have participated in another research study involving an investigational medicinal therapy for ocular disease wit...
AAV-CNGB3 is an investigational gene therapy being developed for achromatopsia, a rare inherited retinal condition. It is designed to deliver a functional copy of the CNGB3 gene to the eye. It has been studied in a Phase 1 clinical trial and is not approved for commercial use.
AAV-CNGB3 is being developed by MeiraGTx Holdings plc, a clinical-stage gene therapy company. The company trades on the Nasdaq under the ticker symbol MGTX. MeiraGTx is responsible for the research, development, and clinical evaluation of this gene therapy candidate.
AAV-CNGB3 has completed a Phase 1 clinical trial. It remains an investigational therapy and has not been approved by the FDA or any other regulatory agency. No active trials are currently listed for this candidate.
AAV-CNGB3 was evaluated in a single Phase 1 clinical trial, NCT03001310, titled Gene Therapy for Achromatopsia (CNGB3). The trial enrolled 23 participants across the United States and the United Kingdom and is now completed. It included participants aged 3 years and older.
Yes, AAV-CNGB3 and AAV - CNGB3 refer to the same investigational gene therapy for achromatopsia. The spacing difference is a formatting variation of the same name. Both refer to the MeiraGTx Holdings plc development program.