Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D01SRB
|
|||
| Former ID |
DIB000342
|
|||
| Drug Name |
AAV2-hRPE65v2
|
|||
| Synonyms |
RPE65 AAV gene therapy (Leber's congenital amaurosis), Children's Hospital of Philadelphia/Second University of Naples; RPE65 adeno-associated viral vector gene therapy (Leber's congenital amaurosis), Children's Hospital of Philadelphia/Second Univerity of Naples
Click to Show/Hide
|
|||
| Indication | Ocular disease [ICD-11: N.A.] | Phase 3 | [1] | |
| Company |
Childrens Hospital of Philadelphia
|
|||
| 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 (NCT00999609) Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis. U.S. National Institutes of Health. | |||
| REF 2 | AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med. 2012 Feb 8;4(120):120ra15. | |||
If You Find Any Error in Data or Bug in Web Service, Please Kindly Report It to Dr. Zhou and Dr. Zhang.