uniQure N.V. is a biotechnology company specializing in gene therapy, focused on developing single-treatment interventions for rare and other severe diseases. Headquartered in Amsterdam, the Netherlands, and founded in 1998, uniQure leverages an adeno-associated virus (AAV) platform to deliver genetic material designed to restore or modulate protein expression in targeted tissues. The company’s commercial activities and partnerships center on AAV-based therapies for liver-directed and central nervous system disorders, including programs in Huntington’s disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and hemophilia. uniQure’s technology platform and internal manufacturing capabilities support research, early clinical development, and late-stage programs, positioning it as a key player in advanced therapies for neurodegenerative and other life-threatening conditions. By focusing on diseases with high unmet medical need and using a modular gene therapy platform, uniQure plays a significant role in the healthcare market segment dedicated to innovative, potentially transformative genetic medicines.