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uniQure N.V. (QURE)

Nasdaq · Manufacturing · Pharmaceutical Preparations

Core research established · 4/4 core data layers currently established

What uniQure N.V. does

​ Overview ​ We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington’s disease, refractory mesial temporal lobe epilepsy (“MTLE”), Fabry disease, and amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (“SOD1-ALS”). ​ Recent Product Candidate Developments ​ Huntington’s disease program (AMT-130) In September 2025, we announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease.

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