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adeno-associated virus vector AAV- CNGA3 · 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 |
|---|---|---|
| Low dose adeno-associated virus (AAV) CNGA3 | EXPERIMENTAL | Subretinal administration of a single low dose AAV CNGA3 |
| Intermediate dose adeno-associated virus (AAV) CNGA3 | EXPERIMENTAL | Subretinal administration of a single intermediate dose AAV CNGA3 |
| High dose adeno-associated virus (AAV) CNGA3 | EXPERIMENTAL | Subretinal administration of a single high dose AAV CNGA3 |
| Name | Type | Description |
|---|---|---|
| adeno-associated virus vector AAV- CNGA3 | BIOLOGICAL | Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene |
Inclusion Criteria: * Are aged years or over * Have achromatopsia confirmed by a retinal specialist investigator Exclusion Criteria: * Are females who are pregnant or breastfeeding * Have participated in another research study involving an investigational medicinal therapy for ocular disease with...
Adeno-associated virus vector AAV-CNGA3 is an investigational gene therapy being studied for the treatment of achromatopsia, a rare inherited retinal disorder that causes color blindness and reduced visual acuity. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
Adeno-associated virus vector AAV-CNGA3 is designed to deliver a functional copy of the CNGA3 gene to retinal cells. The CNGA3 gene encodes a subunit of the cyclic nucleotide-gated channel that is essential for cone photoreceptor function, and mutations in this gene cause achromatopsia.
Adeno-associated virus vector AAV-CNGA3 is being developed by MeiraGTx Holdings plc, a clinical-stage gene therapy company. MeiraGTx is publicly traded on the NASDAQ under the ticker symbol MGTX.
Adeno-associated virus vector AAV-CNGA3 is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA or any other regulatory agency. The drug is still being evaluated for safety and efficacy in clinical trials.
Adeno-associated virus vector AAV-CNGA3 has been studied in one completed Phase 1 clinical trial, identified as NCT03758404. This trial, titled 'Gene Therapy for Achromatopsia (CNGA3)', enrolled 11 participants with achromatopsia and was conducted in the United States and United Kingdom.
Yes, adeno-associated virus vector AAV-CNGA3 is a gene therapy that delivers the CNGA3 gene. It is sometimes referred to as CNGA3 gene therapy in clinical trial descriptions, such as in the trial titled 'Gene Therapy for Achromatopsia (CNGA3)'.