uniQure announced a dosing pause in its Fabry disease AAV gene therapy program after two patients at higher dose levels experienced grade‑3 liver enzyme elevations. The company halted mid‑ and high‑dose cohorts while investigators and regulators review safety signals tied to transaminase elevations. The affected dose included a 4x10^13 gc/kg cohort; uniQure disclosed the events and initiated a safety assessment. The pause mirrors recent gene‑therapy safety scrutiny across AAV programs and will likely delay dose selection and registrational planning. Investors and developers will watch how uniQure and regulators interpret the hepatotoxicity events and what mitigations—steroid management, modified dosing or vector engineering—may be required before resuming higher‑dose arms.