uniQure N.V. (QURE), a gene therapy company developing treatments for severe genetic diseases, announced updated preliminary Phase 1/2a data for AMT-191, its investigational AAV gene therapy for Fabry disease, showing sustained and dose-dependent increases in a-Gal A enzyme activity across all treated patients. The results were presented at the WORLD Symposium in San Diego.
Fabry disease is caused by deficient a-Gal A activity, and current enzyme-replacement therapy requires lifelong infusions. As of the January 8, 2026, cutoff, all 11 patients across three dose cohorts showed elevated a-Gal A levels, with the highest-dose group reaching 27.7-223.7-fold above normal, and the longest-treated patient maintaining supraphysiological expression for over a year.
Six patients have discontinued enzyme-replacement therapy after meeting prespecified criteria, and plasma lyso-Gb3 levels remained stable regardless of ERT status. Safety remained manageable, with no treatment-related serious adverse events at the two lower doses; previously reported events at the highest dose included chest pain, increased troponin, and leptomeningeal enhancement.
uniQure said the results reinforce confidence in AMT-191's biological activity and support its potential as a one-time gene therapy for Fabry disease, with further updates expected as the study continues.
QURE has traded between $7.76 and $71.50 over the past year. The stock is currently trading at $25.78, up 5.14%
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