uniQure halted mid‑ and high‑dose cohorts in its AAV gene‑therapy trial for Fabry disease after two patients experienced grade‑3 liver enzyme elevations. The company reported the safety events and paused doses while it evaluates the events and adjusts monitoring and management strategies. The pause underscores the continuing challenge of dose‑dependent liver toxicity in systemic AAV programs and could affect timelines and dose selection across the field. uniQure’s disclosure also included related program updates and competitor data context. Investors and developers should expect intensified regulatory dialogue and potential protocol amendments for AAV liver safety management.