QURE Profile
uniQure N.V. is a leading gene therapy company dedicated to developing innovative treatments for genetic and other severe diseases. The company focuses on harnessing the power of gene therapy to address unmet medical needs across a range of conditions. Its flagship program, Etranacogene dezaparvovec (AMT-061), is currently in the Phase III HOPE-B pivotal trial, targeting the treatment of hemophilia B. This therapy aims to provide a long-term solution by addressing the root cause of the disease through a single, transformative treatment.
In addition to its work on hemophilia B, uniQure is advancing several other gene therapy programs. AMT-130, which is in Phase I/II clinical trials, targets Huntington's disease, a debilitating neurodegenerative condition. The company is also developing AMT-060, another gene therapy for hemophilia B, currently in Phase I/II trials. Other notable programs include AMT-210 for Parkinson's disease, AMT-260 for temporal lobe epilepsy, and AMT-161 for amyotrophic lateral sclerosis (ALS). AMT-240, still in the preclinical stage, aims to address autosomal dominant Alzheimer's disease.
uniQure’s innovative approach involves using advanced gene therapy techniques to deliver therapeutic genes directly into patients' cells, with the goal of correcting genetic defects and providing long-lasting relief from various genetic disorders. The company’s diverse pipeline reflects its commitment to tackling a wide range of serious health conditions through cutting-edge scientific research and development.
Founded in 1998 and headquartered in Amsterdam, the Netherlands, uniQure N.V. has established itself as a pioneer in the field of gene therapy. The company's continued focus on advancing its clinical programs and expanding its therapeutic portfolio underscores its dedication to improving patient outcomes and transforming the treatment landscape for genetic diseases.
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