Company Profile - UniQure NV

Introduction

UniQure NV (UniQure) develops and markets gene therapy products. The company develops adeno-associated virus (AAV) based gene therapies using its gene technology platform and offers disease-modifying treatments to patients with severe genetic diseases and other devastating diseases. UniQure’s lead product, AMT-060, is a gene therapy for the treatment of moderately-severe hemophilia B. Its preclinical products include AMT-130, an AAV5 vector for the treatment of Huntington's disease and AMT-150 for Spinocerebellar Ataxia type 3. The company develops a gene platform to bring new disease modifying therapies to patients with genetic diseases. The company conducts drug discovery and preclinical research in collaboration with academic research institutions. UniQure is headquartered in Amsterdam, the Netherlands.