Current Research Studies
An Operationally Seamless Phase 1/2/3 Study Consisting of a Safety and Dose-finding Phase 1/2 and Randomized, Open-label, Active-controlled Phase 3 to Evaluate UX701 AAV Gene Therapy in Adults with Wilson Disease
UX701-CL301
What is the goal of the study?
This study investigates the safety and efficacy of a gene therapy (UX701) for Wilson Disease
Who can participate in the study?
Please contact the study team listed below to learn more.