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Funded Studies

Development and Optimization of a Regulatable Gene Switch for Gene Therapies of Parkinson’s Disease

Gene therapy holds tremendous potential for the treatment of Parkinson's disease. By using a virus or other DNA delivery system to permanently insert a new gene into critical regions of a Parkinson patient's brain, it is possible to conceive of novel therapies whereby expression of the new gene may improve patients' response to medication, halt the progression of disease, or even reverse pre-existing damage. Two critical elements for bringing any therapy forward for use in humans are the ability to deliver an effective dose tailored to each patient, and to provide long-term safety. While a number of gene therapies have been shown to be highly effective in animal models of Parkinson's disease, it is considered essential both for safety and for efficacy that clinicians have the capability to "turn on" gene expression at the right level (dose), and to "turn it off" if safety issues are encountered. RheoGene Inc., a biotechnology company based in Philadelphia, has a novel gene regulation technology, the RheoSwitch (R) system, which allows both the level (dose) and timing (on/off) of gene expression to be precisely controlled through the oral administration of a so-called "Activator Drug" in pill form. RheoGene's RheoSwitch (R) system and Activator Drug can be used with any gene and any gene delivery system to provide a safely regulated form of gene therapy. Using the RheoSwitch(R) system, gene therapy expression can be turned on through daily administration of Activator Drug and, in the event of gene toxicity, completely turned off simply through withdrawal of the Activator Drug. MJFF has awarded RheoGene and its collaborator partners a four-year LEAPS award to develop and test the RheoSwitch (R) system and Activator Drug in animal models of Parkinson's disease and Phase I human clinical trials. As part of this LEAPS program, RheoGene has partnered with the NIH Parkinson's Disease Gene Therapy Study Group. The RheoGene team, led by Drs. Dean Cress and Mark Braughler, will optimize the RheoSwitch(R) system for use with different virus delivery platforms and Parkinson's therapy genes, as well as prepare and test the Activator Drug in Phase I human studies. A second team, led by Dr. Martha Bohn at Northwestern University's Children's Memorial Hospital in Chicago, will develop and test the regulated gene therapy virus in rodent models of Parkinson's disease. A third team, led by Dr. Krystof Bankiewicz of the Department of Neurosurgery at UCSF, will test the safety and efficacy of the RheoSwitch (R) system for regulated gene therapy in primates. It is expected that the successful completion of the research sponsored by this LEAPS award will result in a regulated gene therapy that can be safely brought to clinical trials in Parkinson's disease patients.


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