05:04 PM EDT, 10/03/2024 (MT Newswires) -- Ultragenyx Pharmaceutical ( RARE ) said Thursday it saw meaningful clinical activity in the first stage of Cyprus2+ study of UX701 gene therapy for Wilson disease.

The company said six participants out of 15 enrolled tapered off the standard of care treatment among three dose cohorts and it plans to add another cohort with moderately increased dose.

The potential drug also improved copper metabolism.

UX701 has been safe and well-tolerated with no unexpected adverse and significant immunologic safety events as of data cutoff date, Ultragenyx added.

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