uniQure's AMT-191 Gene Therapy Shows Sustained Increases in α-Gal A Activity in Fabry Disease Patients.

uniQure announces initial data from its Phase I/IIa trial of AMT-191, a gene therapy for Fabry disease. All four patients in the first cohort achieved substantial increases in α-Gal A activity, ranging from 27- to 208-fold above the mean normal level. Patients were able to withdraw from enzyme replacement therapy and maintained stable plasma lyso-Gb3 levels. Preliminary data show a manageable safety profile at the highest dose, with updated clinical results expected in the first half of 2026.