Ultragenyx’s Wilson Disease Gene Therapy Program Update

On behalf of the Ultragenyx Wilson disease study team, we would like to share a recent update on our CYPRUS²⁺ study.

Ultragenyx has changed Stage 1 of our CYPRUS²⁺ study to an open-label, single arm design. This means that all patients enrolled in the study will receive UX701, an investigational gene therapy for the treatment of Wilson disease. Stage 1 of the study no longer utilizes placebo, which will allow us to better understand the biological response to UX701 in real-time, as well as optimize the safe reduction of current treatment for WD.

The changes to the CYPRUS²⁺ protocol for Stage 1 may take place at different times depending on site locations. For sites located outside of the U.S., they must await approval by their country’s regulatory agency. Whether or when our investigational gene therapy will be approved by regulatory agencies as a treatment for Wilson disease is not known. The results from additional studies are needed to decide whether this treatment is safe and effective.

You are invited to email PatientAdvocacy@Ultragenyx.com for more information or read the full update located at: https://clinicaltrials.gov/ct2/show/NCT04884815?term=ux701&draw=2&rank=1.