UniQure N.V. is a gene therapy company focused on developing potentially disease-modifying treatments for patients with severe inherited and other serious diseases. Operating as a single business segment, it manages the full lifecycle of its programs—from discovery and development through to commercialization—using a modular gene therapy technology platform.

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UniQure is actively engaged in research and development (R&D) initiatives focused on advancing gene therapy solutions for a range of genetic and acquired diseases. The company's R&D efforts are centered around its proprietary AAV platform, which is primarily directed towards liver and central nervous system disorders. Key programs include the development of AMT-130 for Huntington’s disease, AMT-260 for temporal lobe epilepsy, AMT-191 for Fabry disease, and AMT-162 for ALS caused by SOD1 mutations. UniQure is also exploring new applications for its gene therapy expertise, including potential treatments for Alzheimer’s disease. The R&D activities of UniQure are managed through its research and development unit, which is responsible for the development of product candidates, preclinical research programs, and the advancement of the company's technology platform. This unit oversees the clinical development, regulatory activities, and commercialization efforts related to the company's gene therapy programs. In FY2025, the company spent US$134.4 million on its R&D activities, which declined 4.6% YoY. As of December 2025, UniQure's intellectual property portfolio includes 147 issued patents and 173 pending patent applications. These patents cover a variety of technologies, including product candidates in development and the company's manufacturing and technology platforms.