uniQure N.V.

uniQure N.V. is a biopharmaceutical company specializing in the development of gene therapies for patients with severe genetic and other devastating diseases. Its primary focus lies in leveraging adeno-associated virus (AAV)-based technology to deliver transformative treatments, notably for conditions such as hemophilia B and Huntington's disease. The company has achieved significant milestones, including developing the first approved gene therapy in the Western world and operating the first commercially licensed gene therapy manufacturing facility. uniQure maintains a robust pipeline of clinical-stage gene therapies, collaborating with industry leaders for further development in areas like cardiovascular and neurological diseases. Its business impacts the biotechnology and biopharmaceutical sectors, driving innovation through clinical trials and regulatory advancements. With headquarters in Amsterdam and manufacturing in the U.S., uniQure plays a pivotal role in shaping the future of medical treatment by introducing novel therapeutic approaches for disorders previously lacking effective solutions.