UX701 gene therapy shows clinical activity; 6 of 15 Wilson disease patients tapered off standard-of-care

Ultragenyx Pharmaceutical Inc.

15 patients enrolled across 3 dose cohorts; 6 completely tapered off chelators/zinc therapy; 1 additional began tapering.
Non-ceruloplasmin bound copper stabilized to normal levels; ceruloplasmin-copper activity increased, indicating improved ATP7b function.
UX701 well tolerated with no unexpected treatment-emergent adverse events or significant immunologic safety events.
Company plans to enroll a fourth cohort at moderately increased dose with optimized immunomodulation regimen to enhance efficacy.
Dose selection for randomized placebo-controlled Stage 2 and long-term follow-up in Stage 3 are planned.

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