uniQure N.V., a biopharmaceutical company, develops adeno-associated virus (AAV) based gene therapies through its technology platform for multiple therapeutic areas. The company offers Glybera for the treatment of patients with lipoprotein lipase deficiency. It also develops AMT-060, a gene therapy that is in Phase I/II clinical trial for the treatment of hemophilia B; S100A1, a preclinical therapeutic for congestive heart failure; AMT-021, a gene therapy, which has completed Phase I clinical trial for acute intermittent porphyria; AMT-110, a gene therapy that is in Phase I/II clinical trial for Sanfilippo B syndrome; and AAV2/glial cell line-derived neurotrophic factor for Parkinson's disease. The company has collaborations with Chiesi Farmaceutici S.p.A., Digna Biotech, Institut Pasteur, University of California San Francisco, Bristol-Myers Squibb, and 4D Molecular Therapeutics. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
February 24, 2021
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