• E-mail
• Print
• Comment
• Font Size
• Digg
• del.icio.us
• Discuss article

Fox Foundation to Provide Up to $4.2 Million for Development of Regulatable Gene Therapy Vector

Posted on: Thursday, 5 January 2006, 12:00 CST

NEW YORK, Jan. 5 /U.S. Newswire/ -- The Michael J. Fox Foundation for Parkinson's Research (MJFF) today announced that it would provide up to $4.2 million to a team led by RheoGene Inc. for the development of a gene therapy vector system with a regulatable "on switch" to treat Parkinson's disease. In collaboration with several academic institutions, RheoGene will use the award to develop, optimize and test its "RheoSwitch(r) Therapeutic System" (RTS) technology through Phase I trials within four years. While Parkinson's disease is the first application, the work will have broad applicability to the safety and efficacy of gene therapies for many other diseases as well, and for their accelerated advancement into the clinic.

"This project has the potential to revolutionize the clinical application of gene therapy -- not only for the millions of people with Parkinson's disease, but for countless numbers afflicted by other health ailments as well," said Deborah W. Brooks, MJFF president and CEO. "It is a natural fit with the Foundation's commitment to drive innovative technology that will have a significant impact on patients' lives."

The development of gene therapy as a widespread therapeutic technique has been hampered by the lack of any way to time or finely adjust doses or to "turn off" a gene once it has begun expressing a protein in the brain. The RheoGene-led team will work to use RheoGene's RheoSwitch(r) Therapeutic System (RTS) as a safe and effective means to regulate both the level (dose) and timing of gene expression using an orally administered Activator Drug, or "on switch." The advent of RTS would provide an unprecedented safety mechanism by allowing gene expression to be completely shut off in the event of adverse side effects simply through withdrawal of the RheoGene proprietary Activator Drug.

The project will begin with a focus on two genes. One produces small molecule GDNF, a potent neurotrophic factor; the other produces large molecule AADC, an enzyme involved in dopamine synthesis. Both genes may have therapeutic merit, and different technologies may be required to regulate each. By working on both, the research team can widen the variety of genes to which the regulation switch they develop could ultimately be applicable. Specific milestones built into the award structure will determine whether work on one or both of the genes ultimately goes forward.

The RheoGene award brings MJFF's total commitment to neurotrophic factor research to about $8 million. Neurotrophic factors (also known as trophic or growth factors) have long been considered one of the most promising avenues of Parkinson's research, as they promote survival and improve function of neurons.

"We are very excited that the Fox Foundation has recognized RheoGene's ability to create leading-edge technology," said Thomas Tillett, CEO of RheoGene. "This grant will enable us to continue to build on our successes with the RTS to date in creating innovative solutions that provide safer and more effective gene therapies."

The award is made under the Foundation's LEAPS (Linked Efforts to Accelerate Parkinson's Solutions) initiative. LEAPS are multi-year, multi-million, multi-disciplinary projects to address questions that will have significant practical impact on the understanding and treatment of Parkinson's disease.

The team, led by coordinating principal investigator Dean Cress, Ph.D. and Mark Braughler, Ph.D., of RheoGene, also includes two experts in gene therapy for Parkinson's disease:

-- Krystof Bankiewicz, M.D., Ph.D., professor, Neurological Surgery, and Principal Investigator, Movement Disorders Research Program, University of California, San Francisco

-- Martha C. Bohn, Ph.D., Professor of Pediatrics and of Molecular Pharmacology and Biological Chemistry, Northwestern University, Chicago

About RheoGene Inc.

RheoGene is dedicated to unlocking the power of cell and gene therapy(tm). RheoGene's proprietary RheoSwitch(r) Therapeutic System (RTS) and DirectIt(tm) recombinase platforms are the keys to safe, targeted and regulated cell and gene therapies that deliver the right dose -- to the right place -- at the right time. RheoGene also provides customized RheoSwitch(r) inducible gene expression and AttSite(tm) recombinase products for pharmaceutical discovery and development and biotherapeutics production. RheoGene Inc. is wholly owned by the University of Pittsburgh Medical Center. It has operations within Norristown and Pittsburgh, PA. For more information, visit http://www.rheogene.com.

About The Michael J. Fox Foundation

Founded in 2000, The Michael J. Fox Foundation for Parkinson's Research is dedicated to ensuring the development of a cure for Parkinson's disease within this decade through an aggressively funded research agenda. To date, The Foundation has funded more than $60 million in research aimed at finding a cure for the disease, either directly or through partnerships.

http://www.usnewswire.com

Source: U.S. Newswire

More News in this Category