Company Profile:

Delivering on the promise of gene therapy.

Our focus is to advance the future of medicine through gene therapy.
We have the resources and experience to achieve this.

uniQure is delivering on the promise of gene therapy -- single treatments with potentially curative results.  We are leveraging our 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 other severe genetic diseases.

We are advancing a focused pipeline of innovative gene therapies. We are currently conducting a pivotal phase 3 trial in our lead indication, hemophilia B, and have initiated a phase 1/2 trial in Huntington’s disease.  Our 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. 

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Learn more about our work to deliver curative gene therapies that transform the lives of patients.