Ultragenyx is pleased to announce that the first patient has been dosed in the CYPRUS2+ study, a one-time intravenous (IV) infusion of UX701, an investigational gene therapy for the treatment of Wilson disease. Ultragenyx is sponsoring this global study to determine if UX701 gene therapy is a safe and effective treatment for adults with Wilson disease. This adeno-associated virus (AAV) gene therapy has the potential to restore ATP7B function, which may improve copper metabolism and distribution.
Ultragenyx Update – Feb 17, 2022.
WDA Community Update – Jan 20, 2022.