Early uniQure Fabry Trial Shows Lasting Enzyme Boost, Safety Watch

uniQure N.V. (NASDAQ:QURE) on Friday announced updated preliminary safety and exploratory efficacy data from 11 patients in its Phase 1/2a trial of AMT-191.The updated data was presented at the WORLDSymposium in San Diego, California.Trial DataAMT-191 is an investigational AAV gene therapy under development for Fabry disease, a rare X-linked lysosomal disorder that results in excessive deposition of lipids in the tissues.As of the January 8, 2026, study data cutoff date, all 11 patients in the three dose co ...