uniQure’s sole purpose is to help patients and the people who care for them.
We hope to be a resource for patients about how gene therapy may help.
uniQure is working on delivering on the promise of gene therapy, single treatments with potentially curative results.
See below for an animated demonstration of how gene transfer technology functions within the body:
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.