uniQure

uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular technology platform to rapidly bring new disease-modifying therapies to patients treat patients with CNS, liver/metabolic and cardiovascular diseases.  We are advancing a focused pipeline of innovative gene therapies and have entered late-stage clinical development in our lead indication, hemophilia B, and have established preclinical proof-of-concept in Huntington’s disease.