Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0DX1F
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| Former ID |
DIB000692
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| Drug Name |
Leber's congenital amaurosis gene therapy
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| Synonyms |
LCA gene therapy, Applied Genetic Technologies; Leber's congenital amaurosis gene therapy, Applied Genetic Technologies; RAAV2-CB-hRPE65
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| Indication | Leber congenital amaurosis [ICD-11: 9B70; ICD-10: H35.5] | Phase 1/2 | [1] | |
| Visual disturbance [ICD-11: 9D90; ICD-10: H53] | Phase 1/2 | [2] | ||
| Company |
Applied Genetic Technologies Corp
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Retinal pigment epithelium protein (RPE65) | Target Info | Modulator | [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 | Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA) | |||
| REF 2 | ClinicalTrials.gov (NCT00749957) Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis. U.S. National Institutes of Health. | |||
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