Develops gene therapies for genetic diseases, focusing on treatments for hemophilia B and other rare disorders.
uniQure N.V. is a pioneering gene therapy company dedicated to developing transformative treatments for patients afflicted with genetic and severe diseases. Established in 1998 and headquartered in Amsterdam, the Netherlands, uniQure is at the forefront of advancing therapies that harness the power of genetic medicine.
The company's flagship program, Etranacogene dezaparvovec (AMT-061), is currently in Phase III HOPE-B pivotal trials for the treatment of hemophilia B, demonstrating uniQure's commitment to addressing unmet medical needs in bleeding disorders. Additionally, uniQure is actively developing a diverse pipeline of innovative gene therapies. This includes AMT-130, in Phase I/II clinical trials for Huntington's disease, AMT-060 for hemophilia B, AMT-210 for Parkinson's disease, AMT-260 for temporal lobe epilepsy, AMT-240 for autosomal dominant Alzheimer's disease in preclinical stages, and AMT-161 for amyotrophic lateral sclerosis.
Driven by a mission to bring potentially life-changing therapies to patients worldwide, uniQure leverages cutting-edge research and development to pioneer novel genetic treatments. With a steadfast commitment to scientific innovation and patient care, uniQure N.V. continues to push the boundaries of gene therapy, offering hope to individuals and families affected by devastating genetic conditions.