Our mission is to deliver curative gene therapies that transform the lives of patients.

At uniQure, we are working to bring the future to life.

Imagine a future where a single treatment can replace a lifetime of chronic therapy. A future where one administration can halt the progression of an incurable disorder or add years to the lives of patients. This is the future of gene therapy. 

 See below for an animated demonstration of how gene transfer technology functions within the body:


We hope to be a resource for patients in learning about how gene therapy may help.

In our commitment to rapidly bring new disease-modifying therapies to patients with severe genetic diseases, we are collaborating with Patient Advocacy organizations listed at our community resources page.  Our policy on responding to requests to provide expanded access to our investigational therapies to patients is outlined here.  

We are also committed to keeping you informed on our latest developments, for which you can sign up here. 

Contact Us

For patients:
Daniel Leonard, Senior Director of Global Patient Advocacy

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