QURE, 5-year performance

uniQure (QURE) publishes a study in Gene Therapy

• QURE announces the online publication in Gene Therapy of data demonstrating widespread transduction in the CNS following direct injection of uniQure's AAV5 vector in a large animal model 

 

uniQure announces the online publication in Gene Therapy of data demonstrating widespread transduction in the CNS following direct injection of uniQure's AAV5 vector in a large animal model 
The method of injection used was found to result in very controlled and accurate administration with no adverse events observed in the non-human primates. Varying doses of AAV5 achieved predictable transduction of connected areas of the brain. The data demonstrate that AAV5 is a very effective vector for the central nervous system and has potential for the treatment of a wide range of neurological pathologies. These data will help guide the clinical development of uniQure's gene therapy product candidate AMT-130, which consists of an AAV5 vector carrying an artificial micro-RNA that silences the huntingtin gene for the treatment of Huntington's disease.

uniQure N.V. (QURE) began trading on the NASDAQ on 5 February 2014

The company, based in Amsterdam, develops gene therapies. It raised $91.8 million after offering 5.4 million shares at $17 per share. It will use the money raised to finish building a manufacturing facility in Lexington, Mass., and for clinical trials. Its shares are listed on the Nasdaq under the ticker symbol "QURE." They fell $2.39, or 14.1 percent, to $14.61.

uniQure B.V.
Meibergdreef 61
Amsterdam, 1105 BA
Netherlands

Website: http://www.uniqure.com

Business Summary

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.