Uniqure NV
uniqure.comuniQure NV (NASDAQ: QURE) is a gene therapy company that discovers, develops, and seeks to commercialize treatments for patients with severe genetic diseases. Revenue has been generated through a commercialization and license agreement with CSL Behring LLC, signed June 24, 2020, covering etranacogene dezaparvovec, a gene therapy for hemophilia B. The company operates as a clinical-stage developer with no product sales disclosed in the FY2025 10-K excerpts, carrying interest income of $17.0 million in FY2025, alongside interest expenses of $54.1 million on a royalty financing agreement and $6.9 million on a Hercules debt facility. As of December 31, 2025, uniQure had $50.0 million in outstanding principal under its Hercules loan facility, which has a maximum aggregate amount of $175.0 million. The company underwent significant restructuring beginning in October 2023, eliminating approximately 65% of its global workforce, roughly 300 roles, and sold its Lexington manufacturing facility to Genezen in 2024. Lead pipeline candidate AMT-130 targets Huntington's disease and holds orphan drug exclusivity in the EU.
Products & Services
Etranacogene dezaparvovec: gene therapy for hemophilia B, licensed to CSL Behring LLC. AMT-130: clinical-stage gene therapy candidate for Huntington's disease, granted orphan drug exclusivity in the EU. Gene therapy platform uses adeno-associated virus (AAV) vectors.
Revenue Model
Licensing and collaboration revenue from a commercialization and license agreement with CSL Behring LLC covering etranacogene dezaparvovec (hemophilia B gene therapy); royalty financing agreement with HemB SPV, L.P. (entered May 2023) generating royalty-based obligations; interest income of $16.97 million in FY2025. No product sales revenue is evident from the filing excerpts.
Customers & Markets
CSL Behring LLC is the commercialization partner for etranacogene dezaparvovec. End markets are patients with rare genetic diseases, specifically hemophilia B and Huntington's disease. Healthcare providers, physicians, and third-party payers serve as the prescribing and reimbursement gatekeepers for any approved products.
Geographic Exposure
Incorporated in the Netherlands; operates under EU and U.S. regulatory frameworks. Orphan drug exclusivity for AMT-130 granted in the EU. Subject to U.S. FDA oversight and EU EMA processes. GDPR compliance obligations across EU, UK, and Switzerland.
Financial Snapshot
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