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CLINICAL_TRIALS research · 2026-03-13

Clinical Trial Update: A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease [Phase 1, Phase 2]

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Summary

A Phase 1/2/3 trial of UX701 gene therapy for Wilson disease is currently active but not recruiting.

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Diseases / indications

Drugs mentioned

  • UX701

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: Wilson Disease | Phase 1, Phase 2 | Status: ACTIVE_NOT_RECRUITING