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.

Our platform

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.

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Enabling technologies

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.

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Proprietary manufacturing

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.

Publications

Browse our list of scholarly publications spanning a range of topics, including hemophilia, Huntington’s disease, AAV technology and more.

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Science & Innovation

Spotlight: How AAV Gene Transfer Works

Our platform

Enabling technologies

Proprietary manufacturing

Publications

We want to hear from you.

Science & Innovation

Spotlight: How AAV Gene Transfer Works

Our platform

Enabling technologies

Proprietary manufacturing

Publications

We want to hear from you.

Explore uniQure