AMSTERDAM, THE NETHERLANDS--(Marketwired - Jul 29, 2013) - uniQure B.V., a leader in human gene therapy, today announced the start of a human trial in Parkinson's disease with glial cell line-derived neurotrophic factor (GDNF). The gene therapy developed by researchers at the University of California, San Francisco (UCSF), uses the GDNF gene, which uniQure licensed from Amgen. This trial is part of a wide-ranging collaboration between uniQure and UCSF and represents one of uniQure's suite of GDNF-based gene therapies in development targeting disorders of the central nervous system.

uniQure and UCSF's joint program is an investigator initiated clinical trial in Parkinson's disease, led by Dr. Krystof Bankiewicz MD, PhD, at UCSF, and Dr. John D. Heiss, MD, at the National Institute for Neurological Disorders and Stroke, part of the National Institutes of Health (NIH). Using the ClearPoint Neuro Interventional System, Dr. Bankiewicz's team administered the GDNF gene packaged in an AAV vector to the brain of a first Parkinson's patient. The ClearPoint system has been developed to allow real-time observation of brain surgery, allowing unmatched precision of gene therapy delivery. The hypothesis is that GDNF's neuro-regenerative and protective properties may protect and strengthen brain cells that produce dopamine. Dopamine is a chemical that affects brain function. People with Parkinson's disease have problems producing dopamine in a specific area in the brain responsible for the control of body movements. The study is a Phase I open-label dose escalation safety study that will include 24 patients divided over 4 cohorts. The first patient was dosed on May 20, and so far there have been no safety issues. The study is fully funded by the NIH.

"The start of this trial is the culmination of over 10 years of preclinical work performed at UCSF," says Dr. Krys Bankiewicz of UCSF. "We are extremely proud to have optimized the delivery technologies together with our partners so that we can now safely deliver a gene therapy product into a minuscule space in the brain, including the putamen, observe the surgery in real-time and guarantee the highest possible level of quality and precision of gene delivery. uniQure is our partner of choice for the development of the therapy as they are first in having mastered the vector design, as well as the development and manufacturing challenges gene therapy developers faced in the past."

Under the terms of the collaboration, uniQure will have the rights to the results of the study as well as the IND. In exchange uniQure will manufacture the GDNF-AAV construct using its baculovirus platform for the next phases of the study.

In addition, uniQure is collaborating on a range of GDNF-based CNS disorders, such as multiple system atrophy (MSA), Huntington's disease, and hearing loss together with Universit de Toulouse (France), University of Cambridge (UK), and UCSF. Using AAV5, which has a strong tropism for the CNS, the partners will evaluate these programs up to preclinical proof of concept. As part of this work, uniQure and UCSF are currently developing administration of AAV5 to the cerebrospinal fluid to facilitate broad distribution of substance in the brain.

uniQure has appointed Dr. Bankiewicz chair of its CNS Scientific Advisory Board. Dr. Bankiewicz is the Kinetics Foundation Chair in Translational Research and Professor in Residence of Neurological Surgery and Neurology at UCSF. His research focus is restorative interventions, specifically drug delivery and gene transfer, in models of Parkinson's disease and brain tumors. His laboratory studies delivery of therapeutic agents into the central nervous system through cell-implantation, convection-enhanced and gene transfer-based delivery methods, as well as in vivo applications of MRI and PET to detect in-vivo changes in the brain and their correlation with functional outcome.

"Success in CNS gene therapy is to a large extent dependent on cutting edge delivery technologies. Capitalizing on the unique and broad collaboration with UCSF, in particular the outstanding group in Dr. Bankiewicz' laboratory, we are accessing and developing leading CNS gene delivery technologies. This Parkinson trial also demonstrates the potential of the Company's gene therapy technologies to reach beyond orphan diseases. The modular approach of our manufacturing platform and the use of AAV5 will allow us to rapidly advance a portfolio of promising programs targeting CNS disorders," said Jrn Aldag, CEO of uniQure.

About uniQure uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. Our approach is validated by multiple partnerships and the regulatory approval of our lead product Glybera. http://www.uniqure.com.

DisclaimerThis press release contains forward-looking statements based on uniQure's current expectations. These forward-looking statements include statements regarding the development of a suite of CNS therapies, partnerships in the CNS area, and the development of additional gene therapies. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties regarding further regulatory requirements, the success of further clinical trials, and competitive pressures. uniQure assumes no responsibility to update such forward-looking statements.

Press release (PDF): http://hugin.info/157414/R/1719343/572213.pdf

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uniQure Announces Start of Parkinson's Disease Gene Therapy Phase I Trial by UCSF/NIH to Test Its GDNF Gene in Patients

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