UX701

UX701
	Wilson disease causes a defective copper-binding protein. UX701 is intended to correct the gene producing this protein.
Clinical data
Other names	Rivunatpagene Miziparvovec (USAN US)
Legal status
Legal status	Experimental (as of 2026);
Identifiers
PubChem SID	474492994;
DrugBank	DB18317;

UX701 is an experimental gene therapy for Wilson disease, delivered via adeno-associated virus, that restores a functional version of the ATP7B gene. Wilson disease is a rare genetic disease that results in high levels of copper in the body.

Cyprus2+ study

As of June 2026^[update], UX701 is being evaluated in a Phase 1/2/3 study known as Cyprus2+.

In October 2024, interim data from the first 15 patients showed that 40% had successfully tapered off standard-of-care chelator or zinc therapies while maintaining stable copper levels. Based on these results, Ultragenyx planned to expand the trial with a higher dose and an optimized immunomodulation regimen to further evaluate efficacy, before moving into a dose expansion study.

References

↑ Mazhar, Areej; Piper, Marc S. (October 2023). "Updates on Wilson disease". Clinical Liver Disease. 22 (4): 117–121. doi:10.1097/CLD.0000000000000079. ISSN 2046-2484. PMC 10615495. PMID 37908869.
↑ Zheng, Zi-Wei; Dong, Yi; Wu, Zhi-Ying (September 2022). "Latest innovations in the treatment of Wilson's disease". iLIVER. 1 (3): 181–186. doi:10.1016/j.iliver.2022.09.002. PMC 12212692.
↑ Cataldo, Jason; Allen, Jack; Sankoh, Serap; Weiss, Karl; Askari, Frederick (March 2022). "eP140: A novel, double-blind placebo-controlled seamless phase 1/2/3 AAV9 gene therapy study for Wilson disease". Genetics in Medicine. 24 (3): S86. doi:10.1016/j.gim.2022.01.176.
↑ Ultragenyx Pharmaceutical Inc (2026-06-11). 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 (Report). clinicaltrials.gov.
↑ Stansfield, Noah (October 8, 2024). "In Light of Positive Initial Results, Ultragenyx Seeks to Add Higher Dose Cohort to Trial for Wilson Disease Gene Therapy UX701". CGTlive. MJH Life Sciences. Retrieved March 22, 2026.

[1] Mazhar, Areej; Piper, Marc S. (October 2023). "Updates on Wilson disease". Clinical Liver Disease. 22 (4): 117–121. doi:10.1097/CLD.0000000000000079. ISSN 2046-2484. PMC 10615495. PMID 37908869.

[2] Zheng, Zi-Wei; Dong, Yi; Wu, Zhi-Ying (September 2022). "Latest innovations in the treatment of Wilson's disease". iLIVER. 1 (3): 181–186. doi:10.1016/j.iliver.2022.09.002. PMC 12212692.

[3] Cataldo, Jason; Allen, Jack; Sankoh, Serap; Weiss, Karl; Askari, Frederick (March 2022). "eP140: A novel, double-blind placebo-controlled seamless phase 1/2/3 AAV9 gene therapy study for Wilson disease". Genetics in Medicine. 24 (3): S86. doi:10.1016/j.gim.2022.01.176.

[4] Ultragenyx Pharmaceutical Inc (2026-06-11). 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 (Report). clinicaltrials.gov.

[CGTlive_UX701_2024-5] Stansfield, Noah (October 8, 2024). "In Light of Positive Initial Results, Ultragenyx Seeks to Add Higher Dose Cohort to Trial for Wilson Disease Gene Therapy UX701". CGTlive. MJH Life Sciences. Retrieved March 22, 2026.