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Please Note: The technology listed below is not available to the public at this time. This technology is in the early stage of research and requires further development before it is ready for the marketplace. The VA is currently in the process of identifying potential companies who may be interested in licensing and/or further developing the technology through Cooperative Research and Development Agreements (CRADA). Through cooperative research initiatives such as these, it is our hope and goal that commercial products will be fully developed and made available to benefit veterans and others.
VA TECHNOLOGY OPPORTUNITY BRIEF
Ex vivo and in vivo genetic therapies for bone regeneration
(#04-180, #04-196, #04-204, #07-017)
OPPORTUNITY
The Department of Veterans Affairs (VA) is seeking a commercial partner to license or to further develop gene therapy-based technologies for bone regeneration through a Cooperative Research & Development Agreement (CRADA).
TECHNOLOGY OVERVIEW
Ex vivo gene therapy
The ex vivo gene therapy of this portfolio is designed to stimulate bone synthesis, promote stem cell renewal, and prolong therapeutic efficacy in the treatment of osteoporosis. The therapy also has utility in the rapid healing of bone fractures. Using a novel method for importing recombinant DNA specifically into osteoblast nuclei (#04-204), osteoblast stem cells can be stably transduced with a gene encoding a potent bone-promoting growth factor (#04-196). These modified stem cells can be injected into animals for systemic action. Animal studies to date bode well for potential efficacy in humans.
In vivo gene therapy
As an alternative to systemic treatment with modified stem cells, osteogenic growth factors can be localized to the site of a bone fracture or weakening through retrovirus or lentivirus-based gene therapy (#04-180 & #07-017). Following gene transfer, transduced cells express the modified transgenic osteogenic factor, resulting in high sustained levels at the fracture site and markedly increasing bone synthesis and healing.
BACKGROUND
Ex vivo gene therapy
The ex vivo gene therapy of this portfolio is designed to stimulate bone synthesis, promote stem cell renewal, and prolong therapeutic efficacy in the treatment of osteoporosis. The therapy also has utility in the rapid healing of bone fractures. Using a novel method for importing recombinant DNA specifically into osteoblast nuclei (#04-204), osteoblast stem cells can be stably transduced with a gene encoding a potent bone-promoting growth factor (#04-196). These modified stem cells can be injected into animals for systemic action. Animal studies to date bode well for potential efficacy in humans.
In vivo gene therapy
As an alternative to systemic treatment with modified stem cells, osteogenic growth factors can be localized to the site of a bone fracture or weakening through retrovirus or lentivirus-based gene therapy (#04-180 & #07-017). Following gene transfer, transduced cells express the modified transgenic osteogenic factor, resulting in high sustained levels at the fracture site and markedly increasing bone synthesis and healing.
TECHNICAL MERIT:
Ideal therapies for weak or broken bones would be minimally invasive and of sufficient duration to promote osteogenesis, damaged bone resorption, and angiogenesis (to provide newly deposited tissue with nutrients).
Ex vivo gene therapy
The genetically engineered stem cells of this technology express a growth factor that promotes bone formation and angiogenesis. Furthermore, the stem cells regenerate, providing extended therapy for chronic osteoporosis.
In vivo gene therapy
The osteogenic transgenes in these technologies have been modified for enhanced mRNA stability and protein translation, increasing growth factor expression levels. Use of a retrovirus as a vector for the gene allows targeting to the proliferating periosteal cells that arise shortly after the fracture event. Cellular expression of the transgene results in high sustained levels of the osteogenic growth factor and also promotes angiogenesis.
IP STATUS:
#04-180
U.S. provisional patent application was filed August 11, 2005 (60/707,732).
US non-provisional patent application was filed on August 10, 2006 (11/503,365).
#04-196
US provisional patent application was filed on June 14, 2005 (60/690,696)
A US Non-Provisional patent application was filed on June 13, 2006 (11/452,873).
#07-017
Part of US non-provisional patent application was filed on August 10, 2006 (11/503,365).
#04-204
U.S. provisional patent application was filed April 25, 2005 (60/674,881).
A PCT application was filed on April 24, 2006 (PCT/US2006/015405).
A US Non-Provisional patent application has been filed (S/N not yet assigned).
FOR MORE INFORMATION CONTACT:
Ken Levin, Ph.D.
Technology Transfer Specialist
Technology Transfer Program
Department of Veterans Affairs
Office of Research & Development (12TT)
810 Vermont Avenue, NW
Washington, DC 20420
Phone: 202-461-1713
Fax:202-254-0473
E-mail: Ken.levin@va.gov
Last Updated - July 30, 2008