uniQure N.V. is a clinical-stage biotechnology company specializing in the development and commercialization of gene therapies for patients suffering from serious and often life-threatening diseases. Founded in 1998 as Amsterdam Molecular Therapeutics and rebranded in 2012, uniQure leverages its proprietary adeno-associated virus (AAV) platform to deliver functional copies of genes into patients’ cells. The company’s lead commercial product, etranacogene dezaparvovec (branded Hemgenix®), received regulatory approval for the treatment of adults with hemophilia B, marking uniQure as the first company to bring an AAV-based gene therapy for a bleeding disorder to market.
Beyond hemophilia B, uniQure’s pipeline includes candidates targeting hemophilia A, Huntington’s disease, Fabry disease and Parkinson’s disease, among other rare genetic disorders. The company utilizes its scalable manufacturing infrastructure, which includes facilities in Lexington, Massachusetts and the Netherlands, to produce clinical- and commercial-scale AAV vectors. By maintaining in-house capabilities from vector design to cGMP manufacturing, uniQure aims to streamline development timelines and ensure consistent product quality.
Headquartered in Amsterdam, uniQure serves patients and healthcare providers in the United States, European Union, Canada and other regions where gene therapies have gained regulatory traction. The company’s U.S. subsidiary in Boston supports clinical operations, regulatory engagement and commercial activities, while its European operations coordinate research collaborations and supply logistics. With strategic partnerships and licensing agreements in place, uniQure continues to expand its global footprint and diversify its product offerings.
Under the leadership of Chief Executive Officer Matthew Kapusta, uniQure has strengthened its focus on advancing late-stage gene therapy candidates toward approval and commercialization. Supported by a management team with deep expertise in gene therapy development, manufacturing and regulatory affairs, the company remains committed to addressing unmet medical needs through innovative and durable treatments. As uniQure advances its pipeline and scales its commercial operations, it aspires to transform the standard of care for patients with rare genetic disorders.
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