uniQure N.V. Overview
Pro stress-test →uniQure N.V. is a leading gene therapy company advancing transformative therapies for patients with severe medical needs. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. uniQure is advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases.
Strategic Profile
Pro stress-test →In 2025, uniQure presented compelling 36-month clinical data from AMT-130 demonstrating its potential to become a first disease-modifying therapy for people living with Huntington's disease. However, the FDA reversed its stance on using external controls for AMT-130's approval, creating near-term uncertainty. The company maintains a robust cash position with approximately $622.5 million in cash to support operations into the second half of 2029, with its product pipeline targeting hemophilia, Huntington's disease, and cardiovascular diseases.
Competitive Landscape
Pro stress-test →uniQure developed the first and currently the only gene therapy product to receive regulatory approval in the European Union. The company competes with other gene therapy developers including Solid Therapeutics, Audentes Therapeutics (Astellas), Spark Therapeutics (Roche), and others in rare disease treatment. uniQure's competitive advantage lies in its proprietary AAV manufacturing platform and early regulatory approvals for hemophilia B.
Industry Context
uniQure N.V. operates in Gene Therapy / Biotechnology.
Key facts
Founded: 1998 · Headquarters: Amsterdam, Netherlands · Employees: 209 · Revenue: $16.1M · Market cap: $1.6B