uniQure N.V.

uniQure N.V. is a pioneering biotechnology company dedicated to developing and commercializing innovative gene therapies for patients afflicted with rare and devastating diseases. The company's flagship product, HEMGENIX, represents a significant advancement for individuals living with hemophilia B, offering them the potential to produce their own factor IX protein, thereby reducing the risk of bleeding episodes. Beyond hemophilia B, uniQure is actively advancing a robust pipeline of gene therapy candidates across multiple therapeutic areas. Its lead program, AMT-130, is currently undergoing Phase I/II clinical studies for the treatment of Huntington's disease, a progressive neurodegenerative disorder. Further pipeline assets include AMT-260 for mesial temporal lobe epilepsy, AMT-162 targeting superoxide dismutase enzyme-amyotrophic lateral sclerosis (SOD1-ALS), and AMT-191 for Fabry disease, all in early-stage clinical trials. uniQure leverages its proprietary gene delivery technology and deep scientific expertise to address significant unmet medical needs. The company has established strategic collaborations, including a licensing agreement with Apic Bio for intrathecally administered gene therapy for SOD1-ALS and a development and commercial supply agreement with CSL Behring, underscoring its commitment to advancing its pipeline through partnerships and internal development. Headquartered in Amsterdam, the Netherlands, uniQure is focused on transforming the lives of patients through the power of genetic medicine.