UniQure's AMT-191 Gene Therapy Shows Sustained Efficacy in Fabry Disease Trial

UniQure's AMT-191 gene therapy for Fabry disease has shown sustained and dose-dependent increases in alpha-Gal A enzyme activity in a Phase 1/2a trial. The treatment has elevated alpha-Gal A levels in all 11 patients, with the highest dose reaching 27.7-223.7-fold above normal. Six patients have discontinued enzyme-replacement therapy, and safety has been manageable, with no treatment-related serious adverse events at lower doses. The results support AMT-191's potential as a one-time gene therapy for Fabry disease.