UX701
for Wilson
disease
UX701 (rivunatpagene miziparvovec): Gene therapy for the potential treatment of Wilson disease
Program overview
Stage: Phase 1/2/3
Disease: Wilson disease
Prevalence: 50,000 people in commercially accessible geographies
Disease Mechanism: Inability to normally traffic copper in the body
Symptoms: Liver failure, neurologic deterioration, death
Treatment Modality: AAV9 gene therapy
Treatment Mechanism: Deliver transgene to produce normally functioning ATP7B protein
Wilson disease: Inability to maintain normal copper levels leads to hepatic, neurologic and/or psychiatric problems
Wilson disease is a disorder of copper metabolism caused by mutations in the gene, ATP7B. ATP7B produces a copper-transporter that controls the normal trafficking of copper in the body. Patients with Wilson disease experience hepatic, neurologic and/or psychiatric problems. Those with liver disease can experience such symptoms as fatigue, lack of appetite, abdominal pain and jaundice, and can progress to fibrosis, cirrhosis, life-threatening liver failure and death.
Wilson disease can be treated by reducing copper absorption using zinc or removing excess copper from the body using life-long chelation therapy, but unmet needs exist because some treated patients experience clinical deterioration and severe side effects. An estimated 50,000 people in commercially accessible geographies are affected by Wilson disease.
Evaluating UX701 to properly metabolize copper in tissues and organs
UX701 (rivunatpagene miziparvovec) is an investigational AAV9 gene therapy designed to deliver stable expression of the ATP7B copper transporter following a single intravenous infusion. It has been shown in preclinical studies to normalize copper trafficking and excretion from the body. Ultragenyx has initiated the seamless Cyprus2+ Phase 1/2/3 study of a single intravenous infusion of UX701 in Wilson disease. UX701 leverages Ultragenyx’s Pinnacle PCL™ Platform.
UX701 was granted Orphan Drug Designation in the United States and European Union and Fast Track Designation in the United States.