uniQure NV has announced updated preliminary data from its Phase I/IIa clinical trial of AMT-191, an investigational AAV gene therapy for Fabry disease. The data, presented at the 22nd Annual WORLDSymposium on February 3, 2026, showed sustained and dose-dependent increases in α-Gal A enzyme activity across all dose cohorts, with supraphysiological expression maintained for over a year in the longest treated patient. Six out of eleven patients met criteria to discontinue enzyme replacement therapy, and stable plasma lyso-Gb3 levels were observed post-dosing, regardless of enzyme replacement therapy status. The safety profile remains manageable, with no serious adverse events related to AMT-191 at lower doses, though dosing in the mid- and high-dose cohorts has been paused following asymptomatic liver enzyme elevations in two patients. An oral presentation of the results is scheduled for February 6, 2026, and the presentation will be available on uniQure's website following the event.

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