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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 - UX701-CL301

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Trial Profile

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 - UX701-CL301

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 10 Aug 2026

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At a glance

  • Drugs Rivunatpagene miziparvovec (Primary) ; Eculizumab; Prednisolone; Rituximab; Tacrolimus
  • Indications Hepatolenticular degeneration
  • Focus Adverse reactions; First in man; Registrational; Therapeutic Use
  • Acronyms Cyprus2+
  • Sponsors Ultragenyx Pharmaceutical

Most Recent Events

  • 04 Aug 2026 According to an Ultragenyx Pharmaceutical media release, enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data from this stage are expected in the fourth quarter of 2026.
  • 14 Jul 2026 Status changed from active, no longer recruiting to recruiting.
  • 04 Nov 2025 According to an Ultragenyx Pharmaceutical media release, cohort 4 enrollment complete, data expected in the first half of 2026.

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