Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D04FSD
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| Former ID |
DIB015259
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| Drug Name |
TgAAG76
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| Synonyms |
RAAV 2/2.hRPE65p.hRPE65; AAV RPE65 gene therapy (RPE65-mediated blindness), Targeted Genetics/University of London/Moorfields
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| Indication | Blindness [ICD-11: 9D90] | Phase 1/2 | [1] | |
| Company |
University of London
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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 | ClinicalTrials.gov (NCT00643747) Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis. U.S. National Institutes of Health. | |||
| REF 2 | tgAAG76, an adeno-associated virus delivered gene therapy for the potential treatment of vision loss caused by RPE65 gene abnormalities. Curr Opin Mol Ther. 2010 Aug;12(4):471-7. | |||
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