uniQure 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.

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.

Products and 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.

Customers and end 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.

Value-chain role

Clinical-stage gene therapy developer and IP licensor. Outsourced manufacturing following the sale of its Lexington manufacturing facility to Genezen in 2024. Retains research, clinical development, and regulatory functions.

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.

Source: SEC 10-K, filed 2026-03-02

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