uniQure and Consortium to Receive EUR 2.5
Million Eurostars Grant to Develop RNAi Gene Therapy
for Huntington's Disease

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 Gottingen, Germany, and Maria
Curie-Skłodowska University, Poland.

    (Logo:
http://photos.prnewswire.com/prnh/20130220/595152 )
    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 Jorn 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 [http://www.uniqure.com ] 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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http://photos.prnewswire.com/prnh/20130220/595152
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SOURCE United Business Media