uniQure

uniQure

uniQure launched in 1998 in Amsterdam as a spinout from the Academic Medical Center of the University of Amsterdam, built on the research of co-founder Sander van Deventer. The firm went public on Nasdaq in 2014, shortly after the European Medicines Agency approved Glybera, its lipid disorder treatment that became the first gene therapy approved in a regulated Western market. The founding thesis remains intact: develop adeno-associated virus (AAV) vector-based gene therapies that deliver a corrective gene to address the root cause of disease. Today the firm operates a deep but narrow clinical pipeline grounded in AAV5 vector technology. Its lead program, AMT-130, is a one-time administered gene therapy candidate for Huntington's disease that has been under investigation across multiple Phase I/II trials in the US and Europe. The second pillar is AMT-061, an investigational gene therapy for hemophilia B licensed from Chiesi Group, which has demonstrated durable factor IX expression in late-stage clinical studies. Beyond neurology and hematology, the firm holds preclinical-stage assets primarily targeting liver-directed disorders. The geographic footprint concentrates trial activity in the United States, the Netherlands, and Germany, while the manufacturing supply chain relies on a company-owned facility in Lexington, Massachusetts. As of early 2026, uniQure employed roughly 400 professionals across its Amsterdam headquarters and Lexington manufacturing site. In November 2024, the firm announced a strategic restructuring that reduced its workforce by approximately 30% and narrowed R&D focus to a select set of gene therapy candidates in order to extend its cash runway into 2028 (per Reuters, 2024). The restructuring included the suspension of certain non-core programs and centralized manufacturing operations around the Lexington campus. This move followed the sale of its royalty rights on the hemophilia B gene therapy Hemgenix to a third-party financier in mid-2024, generating substantial non-dilutive capital. uniQure's structural differentiator is its status as a publicly traded, pure-play gene therapy company with full ownership of its own GMP-grade AAV manufacturing infrastructure—a vertical integration model that departs from the contract-manufacturer reliance common among biotech peers. The internal Lexington facility handles vector production from early research through commercial supply, which gives the firm operational control over cost of goods and process consistency. Governance sits with a supervisory board chaired by industry veteran David Meek, while the management board led by CEO Matthew Kapusta maintains the dual Dutch-US corporate structure required for its Nasdaq listing and European scientific roots.