uniQure is focused on delivering on the promise of gene therapy – single treatments with potentially curative results. The company is leveraging its modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and cardiovascular diseases.
uniQure is advancing a focused pipeline of innovative gene therapies and has established clinical proof-of-concept in its lead indication, hemophilia B, and preclinical proof-of-concept in Huntington’s disease. The company’s pipeline of adeno-associated virus (AAV)-based gene therapies has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities. Through recent collaborations and a strategic partnership with Bristol-Myers Squibb to develop gene therapies for cardiovascular diseases, uniQure has taken the next steps toward developing gene therapies targeting chronic and degenerative diseases that affect larger populations.