Details of the Drug

General Information of Drug (ID: DMD1T2D)

Drug Name

UX701

Indication

Disease Entry	ICD 11	Status	REF
Wilson disease	5C64.00	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
Copper-transporting ATPase 2 (ATP7B)	TTOPO51	ATP7B_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 (NCT04884815) A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease. U.S.National Institutes of Health.
2	Clinical pipeline report, company report or official report of Ultragenyx