uniQure halted mid- and high-dose cohorts in its AAV gene therapy trial for Fabry disease after two patients experienced dose-limiting toxicities including grade 3 liver enzyme elevations. The company reported the safety events and paused escalation while investigators and regulators assess causality and patient management. UniQure disclosed the dosing pause and provided preliminary safety details in its clinical update and subsequent filings. The move forces a near-term re-evaluation of the program’s dose strategy and risk mitigation for AAV-mediated systemic delivery, a platform that has produced both approvals and high-profile setbacks across the field. The pause follows other recent gene‑therapy tolerability signals that have prompted sponsors and sites to add more intensive liver monitoring and revised stopping rules. uniQure’s next actions—whether protocol amendments, additional monitoring, or dose-reduction—will shape investor and regulatory confidence in the candidate’s path to later-stage data.