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

General Information of Drug (ID: DMD1T2D)

Drug Name

UX701 Drug Info

Indication

Disease Entry	ICD 11	Status	REF
Wilson disease	5C64.00	Phase 1/2	[1]

Cross-matching ID

Molecule-Related Drug Atlas

Molecule Type:

DTT

Drug Status:

Clinical Trial Drug(s)

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Drug(s) Targeting Copper-transporting ATPase 2 (ATP7B)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
VTX-801	DMQU7HQ	Wilson disease	5C64.00	Phase 2	[3]
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Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Download the Complete Interaction Atlas for Visualization in Cytoscape

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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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
3	ClinicalTrials.gov (NCT04537377) A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients With Wilson's Disease. U.S.National Institutes of Health.