AMSTERDAM, The Netherlands, May 21, 2013 /PRNewswire/ --
uniQure B.V., a leader in the field of human gene therapy, today announced that with its consortium partners it is to receive a EUR 2.5 million Eurostars grant to develop an RNA interference (RNAi) gene therapy for Huntington's disease. The consortium is a pan-European collaboration consisting of uniQure as the coordinator, Lausanne University Hospital, Switzerland, University Medical Center Göttingen, Germany, and Maria Curie-Skłodowska University, Poland.
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The program's aim is to develop a gene therapy for the treatment of Huntington's disease (HD), a rare and devastating neurodegenerative disease caused by mutations in the Huntingtin (Htt) gene. As a result of the defective gene, mutated proteins accumulate in the brain and destroy neurons, leading at first to involuntary, random body movements, but eventually to progressive cognitive decline and finally dementia. Onset of the disease usually manifests itself around 35 to 44 years of age, while life expectancy after diagnosis is on average 20 years. The program will start on June 1, 2013 and run for three years.
"Our Huntington's disease program is part of our strategic effort to demonstrate the potential of our AAV-delivery platform in RNAi," says Jörn Aldag, CEO of uniQure. "The RNAi field has great promise to become a new and important treatment modality. However, the field has been held back by the lack of effective delivery mechanisms. We believe that our AAV technology is ideally suited to deliver RNAi compounds with high accuracy and efficacy. In addition, the development with the consortium of a gene expression system for GDNF will not only benefit the HD program, but also holds great promise for our Parkinson's disease program, and potentially other CNS disorders."
About the program
The consortium's goals are to develop a regulated gene expression system for glial cell derived neurotrophic factor (GDNF) to improve the maintenance and survival of neurons as a HD gene therapy, and to develop regulated expression of artificial miRNA to conditionally silence the Htt gene. The main outcome of the program is a robust pre-clinical assessment of the first regulated gene therapy vector suitable for optimized treatment of HD patients. The ability to regulate gene expression would additionally represent an exciting innovation in the field of gene therapy, creating new opportunities to tackle challenging diseases where gene expression is only required at certain times. The consortium expects that the program's results should lead to clinical safety trials within two years after the conclusion of the project.
About uniQure
uniQure is a world leader in the development of human gene based therapies. uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure's product pipeline of gene therapy products in development comprise hemophilia B, acute intermittent porphyria, Parkinson's disease and Sanfilippo B. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. uniQure's largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at http://www.uniqure.com.
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