UX701

UX701 is an experimental gene therapy for Wilson disease, delivered via adeno-associated virus, that restores a functional version of the ATP7B gene.[1][2][3]

Cyprus2+ Study

UX701 is currently being evaluated in a pivotal Phase 1/2/3 study known as Cyprus2+. In October 2024, interim data from the first three cohorts (15 patients) showed that 6 patients successfully tapered off standard-of-care chelator or zinc therapies while maintaining stable copper levels. Based on these results, Ultragenyx announced plans to add a fourth cohort to Stage 1 of the trial using a higher dose and an optimized immunomodulation regimen to further evaluate efficacy before moving into the Stage 2 dose expansion study.[4]

References

  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. ^ 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.
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