uniQure N.V.

uniQure N.V. is a leading gene therapy company focused on developing and commercializing innovative treatments for severe genetic and other devastating diseases. Headquartered in Amsterdam, Netherlands, the company's mission is to reimagine the future of medicine by delivering potentially curative single-treatment gene therapies. uniQure leverages a proprietary adeno-associated virus (AAV)-based technology platform to advance its pipeline and manufacturing capabilities.

The company's product portfolio and pipeline target significant unmet medical needs. Its flagship approved product is Hemgenix (etranacogene dezaparvovec), a gene therapy for Hemophilia B, which is partnered with CSL Behring. uniQure is also advancing AMT-130, a wholly-owned gene therapy candidate for Huntington's disease, which is in clinical trials, and AMT-260 for refractory mesial temporal lobe epilepsy. Other programs in its pipeline include therapies for Fabry disease and ALS (SOD1).

Recent developments include the FDA granting an accelerated approval pathway for AMT-130 in Huntington's disease in June 2026, with a Biologics License Application (BLA) submission planned for Q3 2026. The company also completed an upsized public offering in June 2026. Matt Kapusta serves as the Chief Executive Officer, leading uniQure's efforts to translate scientific innovation into transformative gene therapies.