uniQure N.V.

AAV gene-therapy pioneer (delivered glybera and AMT-061/HEMGENIX). Strategy now centers on AMT-130, an AAV5 gene therapy for Huntington's disease that posted statistically significant slowing of UHDRS-TFC decline in pivotal Phase 1/2 data and is advancing toward a 2026 BLA. Pipeline expansion targets refractory mesial-temporal lobe epilepsy (AMT-260), Fabry disease (AMT-191), and SOD1 ALS (AMT-162).