Introduced by Bill Wiesler of the Yale Office of Cooperative Research, the speakers were Joseph J. Catino, Ph.D., president and CEO of Helix Therapeutics, and Peter M. Glazer, M.D., Ph.D., who is a Founder of Helix and Professor and Chair of Therapeutic Radiology and Professor of Genetics at Yale University. 

The TFO technology for targeted DNA modifications was discovered in the laboratory of Dr. Glazer. The technology facilitates targeted DNA modifications that can be designed, for example, to correct mutations, alter levels of gene expression, or functionally disrupt molecules critical to disease processes. The Helix approach does not rely on viral vectors and corrects the patients’ own blood stem cells, providing stable, heritable changes through autologous cell therapy.

Through the use of SBIR grants and seed funding, Helix plans to advance preclinical projects targeting the human globin gene for applications on sickle cell anemia and beta-thalassemia, the CCR5 receptor for the HIV/AIDS population, and genetic mutations responsible for lysosomal storage disease. The current mission is targeting blood-based genetic diseases; however, the company envisions applications in a broad array of human genetic diseases as advances in human stem cell biology are realized.