uniQure (NASDAQ:QURE) is a biotechnology company focused on the development and commercialization of gene therapies for patients with severe medical needs. Using its proprietary adeno‐associated viral (AAV) vector platform, the company designs single‐dose treatments aimed at addressing the underlying genetic causes of disease rather than solely managing symptoms. Its most advanced program, Hemgenix® (etranacogene dezaparvovec), received regulatory approval in the United States and Europe for adult patients with hemophilia B, marking one of the first gene therapies for a bleeding disorder to reach the market.
Beyond hemophilia B, uniQure’s pipeline includes preclinical and clinical-stage candidates targeting rare and debilitating conditions such as aromatic l-amino acid decarboxylase (AADC) deficiency, Huntington’s disease, and Parkinson’s disease. The company leverages its scalable manufacturing capabilities and in-house process development teams to optimize vector production and ensure supply chain robustness. Through strategic partnerships and collaborations, uniQure also explores ex vivo approaches for oncology and the engineering of next-generation AAV capsids to broaden its therapeutic reach.
Founded in 1998 as a spin-off from academic research in the Netherlands, uniQure has built a transatlantic presence with its corporate headquarters in Lexington, Massachusetts, and its European research and manufacturing operations in Amsterdam. This geographic footprint enables the company to engage with leading clinical centers and regulatory authorities across North America and Europe, while advancing global registration strategies for its therapies.
The company’s leadership team is led by President and Chief Executive Officer Jeroen Louwerse, who brings extensive experience in gene and cell therapy development. Under his guidance, uniQure continues to invest in research and manufacturing infrastructure and to expand its scientific collaborations. The company remains committed to delivering durable, one-time treatments that have the potential to transform the standard of care for patients with serious genetic disorders.
