Success Stories

UT-Battelle Licenses Treatments for Human Diseases

While  a researcher at ORNL, Dr. Cymbeline Culiat of NellOne Therapeutics, Inc., identified the role of the Nell1 gene and its signaling pathway in the formation of bone, cartilage, skeletal muscle, and cardiac muscle—a discovery that was the basis for patents for applications of the Nell1 gene and protein in diagnostics and treatments for certain human diseases. UT-Battelle, which manages ORNL for DOE, has such applications covering the Nell1 system in the three treatment areas of cartilage and vertebral disc growth, cardiac repair, and skeletal muscle regeneration. Now it is partnering with NellOne Therapeutics, a startup company and spin-out from ORNL, to commercialize two of these treatments. Recently it exclusively licensed NellOne Therapeutics patents for the Nell1 system for the fields of cardiology and skeletal muscle regeneration.

Dr. Culiat’s discoveries at the laboratory were based on studies of a unique mutant mouse stock generated from a large-scale mutagenesis program funded by DOE. ORNL pioneered the techniques for generating the single base mutations in the mouse genome that enabled the Nell1 research. Moreover, the laboratory had comprehensive resources for conducting genetic research that included a state-of-the-art pathogen-free mouse facility, molecular biology and genomics laboratories (for microarray and genotyping), and more than five decades of scientific expertise in mammalian genetics, all of which were used in building the Nell1-based technology.   

NellOne Therapeutics is a regenerative medicine company focused on protein therapeutics for restoring cardiac muscle mass and function in patients recovering from heart attacks and for regenerating skeletal muscle. It has an initial $1.5 million seed investment from Battelle Ventures and IVP. For more information, see www.nell-one.com.