Date: 2013-07-29
Type of information:
phase: 1
Announcement: initiation of the study
Company: Uniqure (The Netherlands) University of California, San Francisco (UCSF) (USA)
Product: glial cell line-derived neurotrophic factor (GDNF)-based gene therapy
Action mechanism: gene therapy
Disease: Parkinson\'s disease
Therapeutic area: Neurodegenerative diseases - CNS diseases
Country: USA
Trial details: The study is a Phase I open-label dose escalation safety study that will include 24 patients divided over 4 cohorts.
Latest
news: * On July 29, 2013, uniQure, a leader in human gene therapy, has 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.
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.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 Université 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.
