UniQure paused mid- and high-dose cohorts in its AAV gene‑therapy study for Fabry disease after patients developed dose-limiting liver toxicities. The company reported grade‑3 liver enzyme elevations at a 4x10^13 gc/kg dose and moved to suspend escalation while monitoring affected participants and reviewing safety data. Regulators and investors will watch the company’s safety-run decisions closely because AAV hepatotoxicity has become a recurring barrier for systemic gene therapies. The pauses follow internal safety reviews and mirror wider concerns about vector dosing and liver adverse events across the field. UniQure said it is evaluating next steps for dose selection and patient management; the episode underscores how even small numbers of severe adverse events can prompt rapid protocol changes in life‑saving rare‑disease programs. Clinicians should note that the pause affects ongoing enrollment and timelines for pivotal plans.