uniQure N.V. QURE

Company Overview

uniQure N.V. is a biotechnology company focused on the discovery, development, and commercialization of gene therapies based on adeno-associated viral (AAV) vectors. The company operates within the biotechnology and gene therapy industries, targeting severe genetic and neurological diseases with high unmet medical need. Its core competency lies in developing one-time, potentially curative treatments by delivering functional genes to patients.

The company’s primary value drivers include its proprietary AAV-based gene therapy platform and a pipeline led by therapies for hemophilia B and Huntington’s disease. uniQure is uniquely positioned as one of the first gene therapy companies to achieve regulatory approval for a systemically delivered AAV gene therapy. The company was founded in 1998 in the Netherlands as Amsterdam Molecular Therapeutics, later rebranded as uniQure, and became publicly listed on NASDAQ in 2014.