Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DMFHV9L)

• Drug Name: Leber's congenital amaurosis gene therapy Drug Info
• 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 Drug ID: DMFHV9L

Molecule-Related Drug Atlas

Drug(s) Targeting Retinal pigment epithelium protein (RPE65)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
AAV2-hRPE65v2	DMJNSLU	Ocular disease	1F00.1Z	Phase 3	[3]
Emixustat	DMH8VMP	Age-related macular degeneration	9B75.0	Phase 2/3	[4]
TgAAG76	DMNKLZM	Blindness	9D90	Phase 1/2	[5]
AAV-RPE65	DMAH6UJ	Leber congenital amaurosis	9B70	Phase 1/2	[6]

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]

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