uniQure receives FDA RMAT designation for gene therapy AMT-130 in Huntington's disease

uniQure N.V.

FDA granted RMAT designation for AMT-130 based on 24-month Phase I/II data vs. natural history cohort.
First RMAT designation ever for a Huntington's disease therapy, enabling fast track and breakthrough therapy benefits.
Designation allows increased collaboration with FDA on accelerated approval pathways and expedited development.
uniQure expects to provide updated interim data from Phase I/II studies in mid-2024, including up to 3-year follow-up on 29 patients.

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