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 developed by MeiraGTx Holdings plc for Leber Congenital Amaurosis, a rare inherited retinal disease. It uses an adeno-associated virus (AAV) vector to deliver the RPE65 gene. It is in Phase 1 clinical development and has been studied in a single completed trial.
AAV RPE65 is being developed for the treatment of Leber Congenital Amaurosis, a rare genetic eye disorder that causes severe vision loss. The therapy is intended to address the underlying RPE65 gene defect. It is investigational and has not been approved for any indication.
AAV RPE65 is developed by MeiraGTx Holdings plc, a clinical-stage gene therapy company. The company's ticker symbol is MGTX. MeiraGTx is responsible for the research, development, and clinical testing of this gene therapy candidate for Leber Congenital Amaurosis.
AAV RPE65 is in Phase 1 clinical development. It has completed one Phase 1 trial with 15 participants. The trial was controlled but not randomized or double-blind. The therapy remains investigational and has not been approved by the FDA or any other regulatory agency.
AAV RPE65 has been studied in one completed Phase 1 clinical trial, NCT02781480, titled "Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)." The trial enrolled 15 participants across the United States and the United Kingdom, with a minimum age of 3 years.