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 developed for the treatment of Leber Congenital Amaurosis, a rare inherited retinal disease. It is designed to deliver a functional copy of the RPE65 gene to retinal cells in patients affected by this condition.
AAV RPE65 is being developed by MeiraGTx Holdings plc, a clinical-stage gene therapy company. The company trades on the Nasdaq under the ticker symbol MGTX.
AAV RPE65 is in Phase 1 clinical development. It is an investigational gene therapy and has not been approved by the FDA or any other regulatory agency. The Phase 1 trial has been completed.
AAV RPE65 has been evaluated in one clinical trial, NCT02781480, titled "Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)." This Phase 1 trial enrolled 15 participants across the United States and the United Kingdom and is now completed.
AAV RPE65 is a gene therapy that uses an adeno-associated virus (AAV) vector to deliver the RPE65 gene to retinal cells. The RPE65 gene encodes a protein essential for the visual cycle, and restoring its function is intended to address the underlying cause of Leber Congenital Amaurosis.