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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 studied for the treatment of achromatopsia, a rare inherited retinal disorder that causes color blindness and poor visual acuity. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
AAV-CNGB3 is designed to deliver a functional copy of the CNGB3 gene to retinal cells. The CNGB3 gene provides instructions for a subunit of a cyclic nucleotide-gated channel essential for normal cone photoreceptor function, and mutations in this gene cause achromatopsia.
AAV-CNGB3 is being developed by MeiraGTx Holdings plc, a clinical-stage gene therapy company traded on the Nasdaq under the ticker symbol MGTX. The company is focused on developing transformative gene therapies for serious diseases.
AAV-CNGB3 is in Phase 1 clinical development. A Phase 1 trial has been completed, and the therapy remains investigational. It has not received FDA approval or any other regulatory approval for commercial use.
AAV-CNGB3 has been studied in one completed Phase 1 clinical trial, registered as NCT03001310, titled "Gene Therapy for Achromatopsia (CNGB3)." The trial enrolled 23 participants with achromatopsia and was conducted in the United States and the United Kingdom.
AAV-CNGB3 is a specific gene therapy that delivers the CNGB3 gene. Other gene therapies for achromatopsia may target different genes, such as CNGA3, which also causes the condition. AAV-CNGB3 is distinct in its genetic target and is not interchangeable with other investigational therapies.