Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DDX95P
|
|||
| Drug Name |
AAV-RPE65
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Leber congenital amaurosis [ICD-11: 9B70; ICD-10: H35.5] | Phase 1/2 | [1] | |
| Company |
MeiraGTx
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Retinal pigment epithelium protein (RPE65) | Target Info | . | [2] |
| BioCyc | The visual cycle I (vertebrates) | |||
| KEGG Pathway | Retinol metabolism | |||
| Pathwhiz Pathway | Retinol Metabolism | |||
| Pathway Interaction Database | Visual signal transduction: Cones | |||
| Visual signal transduction: Rods | ||||
| Reactome | The canonical retinoid cycle in rods (twilight vision) | |||
| WikiPathways | Vitamin A and Carotenoid Metabolism | |||
| Visual phototransduction | ||||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT02781480) Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65). U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of MeiraGTx. | |||
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