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uniQure N.V. Description

uniQure BV, a biopharmaceutical company, develops gene therapies through its modular technology platform for the treatment of genetic or acquired diseases. The company offers Glybera for the treatment of patients with lipoprotein lipase deficiency, an orphan metabolic disease. It also develops a pipeline of additional adeno-associated virus (AAV) based gene therapies, including AMT-060, a gene therapy for the treatment of hemophilia B; AMT-021 for acute intermittent porphyria; AMT-110 for Sanfilippo B syndrome; and AAV2/glial cell line-derived neurotrophic factor for Parkinson's disease, as well as various programs that are in early preclinical development stage. The company has collaborations with Chiesi Farmaceutici S.p.A., Digna Biotech, Institut Pasteur, and University of California San Francisco. uniQure BV was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

Sector: Healthcare
Industry: Biotechnology
Keywords: Biopharmaceutical Biology Disease Clinical Development Gene Therapy Genetics Parkinson's Disease Metabolic Disease Applied Genetics Gene Delivery Lipoprotein Hemophilia Adeno Associated Virus Treatment Of Hemophilia Acute Intermittent Porphyria Alipogene Tiparvovec Glybera Lipase Lipoprotein Lipase Deficiency