A Phase 1/2/3 Study of UX701 Gene Therapy in Adults with Wilson Disease”.

You likely can't join if

• 1.Detectable pre-existing antibodies to the AAV9 capsid (AAV9 DetectCDx). 2.Stage 1 only: History of copper chelator or zinc therapy noncompliance, in the Investigator’s judgment, within 6 months prior to Screening. 3.History of liver transplant. 4.Active decompensated hepatic cirrhosis or history of hepatic encephalopathy. 5.Significant hepatic inflammation as evidenced by any of the following laboratory abnormalities 6.Model for End-Stage Liver Disease (MELD) score > 13. 7.Hemoglobin < 9 g/dL. 8.Presence of Stage 3 or higher chronic kidney disease based on estimated glomerular filtration rate < 60 mL/min/1.73 m2. 9.Marked neurological deficit or compromise that, in the Investigator’s opinion, would interfere with the subject’s safety or ability to participate in the study. 10.Moderate to severe depression, recent or active suicidal ideation with intent or suicidal behavior, psychosis, or unstable psychiatric illness 20.Known hypersensitivity to UX701 or its excipients, copper chelators (ie, penicillamine, trientine), zinc, rituximab, tacrolimus, corticosteroids, or eculizumab that, in the Investigator’s judgment, places the subject at increased risk for adverse effects. 21.Participation in another gene transfer study or use of another gene transfer product before or during study participation. 24.Subjects with known hypersensitivity to amide-containing local anesthetics are excluded from participating in the optional liver biopsy substudy.