In a major step toward commercialization of a promising therapeutic treatment, Oak Ridge National Laboratory contractor UT-Battelle has exclusively licensed patents on inventions based on the Nell-1 gene to NellOne Therapeutics, Inc. (NellOne), a company spun out of the Department of Energy laboratory.
The protein therapy treatment under development takes advantage of the Nell-1 gene's cell-signaling pathway that controls tissue growth and maturation in mammalian organs.
The foundation for this therapy is research performed by Cymbeline Culiat, who as an ORNL systems genetics researcher identified the role that the Nell-1 pathway plays in tissue growth and maturation.
Today, Culiat is leading the NellOne research effort to translate the Nell-1 pathway discoveries into a therapy that restores both mass and function to damaged human tissues, such as heart and skeletal muscle.
If successful, the protein therapy could improve the lives of victims of heart attacks and severe muscle wounds. Other therapies, such as stem-cell treatments, have succeeded in triggering tissue formation but fall short in restoring the actual function of the tissue.
Battelle Ventures, with its Knoxville-based affiliate fund, Innovation Valley Partners (IVP), created NellOne with a $1.5-million seed investment in 2008.
"Our executing this license is confirmation from NellOne that sufficient proof-of-principle experiments have been completed and that the company is progressing toward the commercialization of an extremely promising technology that could one day vastly improve the lives of countless heart patients," said Tracy Warren, NellOne chief executive officer and Battelle Ventures general partner.
"NellOne is currently dedicated to the development of intellectual property and moving into a clinical setting," Warren continued, noting that it operates out of the IVP office in Knoxville. "A medical treatment based on the patented technologies is years away, making Battelle Ventures' support even more critical."
"This licensing agreement is a statement not only of NellOne's promise, but also of how vital the support of venture capital investment is during these crucial early years of technology development and nurturing," said ORNL Partnerships Director Tom Ballard. "This announcement is an important step along the way to the marketplace past the initial proof-of-concept stage."
Battelle Ventures and IVP have a combined $255 million—$220 million and $35 million, respectively—to create and accelerate early-stage technologies, including in the East Tennessee region. The original research on Nell-1 was supported by the DOE Office of Science.