rivunatpagene miziparvovec

UX701 (rivunatpagene miziparvovec) is an investigational gene therapy developed for the treatment of Wilson disease, a rare genetic disorder caused by mutations in the ATP7B gene that disrupt copper metabolism. The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the ATP7B gene via a single intravenous infusion. This approach aims to restore normal copper trafficking and excretion by enabling stable expression of the ATP7B copper transporter protein in patients’ tissues. Preclinical studies have shown normalization of copper metabolism, and early clinical data indicate improved copper handling and reduction or discontinuation of standard-of-care chelation therapies in some patients. UX701 is currently being evaluated in a global Phase 1/2/3 clinical trial (CYPRUS2+), with orphan drug designation granted in both the US and EU, as well as Fast Track status from the FDA[1][2][4][5][6].

Brand names

UX701UX-701UX 701

Other names

rivunatpagene miziparvovec