Details of the Drug

General Information of Drug (ID: DMAH6UJ)

Drug Name

AAV-RPE65

Indication

Disease Entry	ICD 11	Status	REF
Leber congenital amaurosis	9B70	Phase 1/2	[1]
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Drug Type

Gene therapy

Cross-matching ID

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	Not Available	[2]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT02781480) Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65). U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of MeiraGTx.