uniQure Reports Updated Preliminary Data for AMT-191 Gene Therapy in Fabry Disease Patients

uniQure has announced updated preliminary data from its Phase I/IIa trial of AMT-191, a gene therapy for Fabry disease. The data showed sustained increases in α-Gal A activity in patients across all three dose cohorts, with six patients discontinuing enzyme replacement therapy. The highest dose resulted in α-Gal A activity ranging from 27.7- to 223.7-fold above mean normal levels. Stable lyso-Gb3 levels were maintained post-dosing, regardless of enzyme replacement therapy status. The study remains ongoing, and the company believes the data supports the potential of AMT-191 as a one-time administered gene therapy for Fabry disease.