General Information of DTT (ID: TTBOH16)

DTT Name Retinal pigment epithelium protein (RPE65) DTT Info
Gene Name RPE65

Molecule-Related Drug & Molecule List

Molecule-Related Drug List
Molecule Type:
DTT
DTP
DME
Drug Status:
Clinical Trial Drug(s)
5 Clinical Trial Drug(s) Transported by This DTP
Drug Name Drug ID Indication ICD 11 Highest Status REF
AAV2-hRPE65v2 DMJNSLU Ocular disease 1F00.1Z Phase 3 [1]
Emixustat DMH8VMP Age-related macular degeneration 9B75.0 Phase 2/3 [2]
AAV-RPE65 DMAH6UJ Leber congenital amaurosis 9B70 Phase 1/2 [3]
Leber's congenital amaurosis gene therapy DMFHV9L Leber congenital amaurosis 9B70 Phase 1/2 [4]
TgAAG76 DMNKLZM Blindness 9D90 Phase 1/2 [5]
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Molecule Interaction Atlas

References

1 AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med. 2012 Feb 8;4(120):120ra15.
2 Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
3 Clinical pipeline report, company report or official report of MeiraGTx.
4 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.
5 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.