Science & Innovation
Gene therapy leadership in constant motion.
From our leading AAV platform and enabling technologies to our proprietary manufacturing capabilities, we are working to change the future of medicine through gene therapy.
Focused primarily on liver-directed and central nervous system disorders, our best-in-class AAV delivery system is showing exciting potential in an increasing number of genetic or acquired diseases.Learn More
From next-generation capsids to new platforms for targeted delivery and dosing, uniQure is developing a growing array of technologies to enable next-level gene therapy efficacy, biodistribution and safety.Learn More
Our facility in Lexington, Massachusetts, applies more than two decades of gene therapy manufacturing expertise to provide highly scalable, flexible production capabilities to support uniQure programs.Learn More
Browse our list of scholarly publications spanning a range of topics, including hemophilia, Huntington’s disease, AAV technology and more.Learn More
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