UniQure said it paused mid‑ and high‑dose cohorts in its Fabry disease gene therapy study after two patients who received 4x10^13 gc/kg experienced grade 3 liver enzyme elevations. The safety signal prompted an immediate halt to higher doses while the company evaluates causality and next steps. UniQure disclosed the pause amid a broader gene‑therapy field that has tightened pharmacovigilance for dose‑related hepatic events. The dose suspension may delay development timelines and will be closely watched by investors and partners given the commercial promise of durable gene therapies for rare lysosomal disorders.
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