Description
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, spinocerebellar ataxia, 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.