Details of the Drug

General Information of Drug (ID: DMJNSLU)

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
Indication
Disease Entry ICD 11 Status REF
Ocular disease 1F00.1Z Phase 3 [1]
Cross-matching ID
TTD ID
D01SRB

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 ClinicalTrials.gov (NCT00999609) Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis. U.S. National Institutes of Health.
2 AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med. 2012 Feb 8;4(120):120ra15.