uniQure doses first patient in Phase I/IIa AMT-191 Fabry disease trial

uniQure N.V.

First patient dosed in open-label, two-cohort Phase I/IIa trial of AMT-191 gene therapy for Fabry disease.
Low-dose cohort: 6e10^13 gc/kg; high-dose: 3e10^14 gc/kg; one-time IV infusion; 24-month follow-up.
AMT-191 uses same AAV5 delivery as HEMGENIX; targets liver to produce GLA protein.
CEO cites catalysts: FDA engagement for AMT-130 (Huntington's), new trials in epilepsy and SOD1-ALS.
Cash runway extended through end of 2027 from cost reduction initiatives.

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