Sponsor: RheoGene, Inc.

• Project duration: 10/1/2004 - 9/30/2007
• Total project (est.): $2,496,968
• Requested ATP funds: $1,997,574

Many clinical uses are envisioned for stem cells - precursor cells that develop into various mature cells for the development, regeneration, and renewal of body tissues. In particular, scientists' ability to isolate and manipulate neural stem cells in vitro has generated hope for genetically engineering and transplanting these cells to supplant neural cells that have been damaged or lost in central nervous system disorders such as Alzheimer's disease and spinal cord injuries. However, the clinical use of adult stem cells has been delayed by health risks and technical challenges. It is not yet possible to predictably insert genes in the right places in chromosomes, eliminate misplaced genes, deactivate cancer genes, or regulate gene behavior over the long term. Rheogene Inc. plans to develop new techniques for site-specific insertion and control of genes in human adult neural stem cells, to enable the production of proteins to treat incurable ocular diseases. Neural stem cells differentiate into several different types of nervous system cells. The company will develop the technologies for extending the life span of neuronal stem cells, inserting genes at specific locations in the chromosomes of adult stem cells, and controlling the production timing and levels of therapeutic proteins made by several different genes in stem cells. In addition, a "safety switch" will be developed to eliminate the engineered cells if problems develop, such as activation of a cancer gene. The highly engineered neuronal stem cells will be tested in mouse models for retinal and optic nerve disease, the major causes of incurable vision loss in developed countries. Children's Hospital of Orange County (Orange, Calif.) will supply the human neural stem cells, assist in other aspects of basic research, and conduct animal studies. ATP funding will broaden the scope of the project to focus on a platform technology and is needed because the research is at too early a stage to attract venture capital or commercial investors. If successfully developed, the new technology will substantially improve the safety, reliability, and effectiveness of stem cell therapies. The technology could be used to make stem cell preparations for transplant to treat ocular diseases, potentially reducing the $67 billion annual direct and indirect costs attributed to vision impairment and blindness. The technology also has broad potential for treating other serious, chronic conditions such as central nervous system disorders and cardiovascular diseases.

For project information:

Lorraine Keller, Ph.D, (610) 650-8734
lkeller@rheogene.com

ATP Project Manager

Michael Amos, 301-975-8631
michael.amos@nist.gov

This is the fact sheet for this project as it was announced on September 28, 2004.
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