UniQure NV (UniQure) provides gene therapy-based treatments. The company offers disease-modifying treatments through its modular technology platform to patients with severe genetic diseases and other devastating diseases. The company operates its business as a single segment, which comprises the discovery, development, and commercialization of innovative gene therapies.

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The company also conducts research to advance various other gene therapy candidates targeting rare and orphan diseases and genetic diseases that affect liver, including hemophilia A, and various CNS disorders. The company also focuses on developing therapies for cardiovascular diseases. In FY2022, the company incurred R&D expense of US$197.6 million. The company had 120 patents and 30 patents in U.S. and 14 European and 160 pending patent applications.