Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DCY10V
|
|||
| Drug Name |
UX701
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Wilson disease [ICD-11: 5C64.00] | Phase 1/2 | [1] | |
| Company |
Ultragenyx Pharmaceutical
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Copper-transporting ATPase 2 (ATP7B) | Target Info | . | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 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. | |||
| REF 2 | Clinical pipeline report, company report or official report of Ultragenyx | |||
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