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]
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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]

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Molecular Interaction Atlas (MIA)

MIA Detail

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.