Details of the Drug

General Information of Drug (ID: DMFHV9L)

Drug Name
Leber's congenital amaurosis gene therapy
Synonyms LCA gene therapy, Applied Genetic Technologies; Leber's congenital amaurosis gene therapy, Applied Genetic Technologies; RAAV2-CB-hRPE65
Indication
Disease Entry ICD 11 Status REF
Leber congenital amaurosis 9B70 Phase 1/2 [1]
Visual disturbance 9D90 Phase 1/2 [2]
Cross-matching ID
TTD ID
D0DX1F
Repurposed Drugs (RPD) Click to Jump to the Detailed RPD Information of This Drug

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Retinal pigment epithelium protein (RPE65) TTBOH16 RPE65_HUMAN Modulator [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
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.