Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AAV RPE65 · 1 trial · 1 indication
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more. * Severe unresponsive inflammation. * Infective endophthalmitis. * Ocular malignancy. * Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
| Arm | Type | Description |
|---|---|---|
| Low dose AAV-RPE65 | EXPERIMENTAL | Subretinal administration of a single low dose of range AAV-RPE65 |
| Intermediate dose AAV-RPE65 | EXPERIMENTAL | Subretinal administration of a single intermediate dose of range AAV-RPE65 |
| High dose AAV-RPE65 | EXPERIMENTAL | Subretinal administration of a single high dose of range AAV-RPE65 |
| Name | Type | Description |
|---|---|---|
| AAV RPE65 | BIOLOGICAL | Comparison of different dosages of AAV RPE65 |
Key Inclusion Criteria: * Aged 3 years or older * Early-onset severe retinal dystrophy consistent with RPE65 deficiency Key Exclusion Criteria: * Females who are pregnant or breastfeeding * Have participated in another research study involving an investigational therapy for ocular disease within ...
AAV RPE65 is an investigational gene therapy being studied for the treatment of Leber Congenital Amaurosis (LCA), a rare inherited retinal disease. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
AAV RPE65 is being developed by MeiraGTx Holdings plc, a biopharmaceutical company traded on the Nasdaq under the ticker symbol MGTX. The company is conducting clinical research to evaluate the therapy for Leber Congenital Amaurosis.
AAV RPE65 is in Phase 1 clinical development. It is an investigational therapy, meaning it has not yet received regulatory approval and is still being evaluated for safety and efficacy in clinical trials.
AAV RPE65 has been studied in a completed Phase 1 clinical trial with the identifier NCT02781480. The trial, titled 'Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA),' enrolled 15 participants and was conducted in the United States and United Kingdom.
No, AAV RPE65 is not a monoclonal antibody. It is a gene therapy being developed for Leber Congenital Amaurosis. The distinction is important for understanding its mechanism and how it is administered in clinical trials.