RARE biotech catalysts & pipeline

ULTRAGENYX PHARMACEUTICAL INC · Data as of 2026-10-19

Upcoming catalysts

rivunatpagene miziparvovec — Clinical data readout

Q4 2026

• UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for the treatment of Wilson disease: 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.

Source filing

avalotcagene ontaparvovec — Topline data

H1 2027

• DTX301 (avalotcagene ontaparvovec) AAV8 gene therapy for the treatment of Ornithine Transcarbamylase, or OTC, deficiency: The Phase 3 Enh3ance study continues with patients in both treatment and cross-over groups progressing through 64 weeks of follow-up. Data from the second primary endpoint, which evaluates reduction in treatment burden, including use of ammonia scavengers and dietary management, are expected in the first half of 2027.

Source filing

Explore the free biotech catalyst calendar