Drive for Diagnosis 2024 Golf Classic

TGEN Collaboration

The Translational Genomics Research Institute (TGen), part of City of Hope, is a nonprofit biomedical research organization where scientists conduct groundbreaking research with lifechanging results. Within its Center for Rare Childhood Disorders (the Center), our faculty decipher a patient’s genome to generate essential information on that patient’s genetic makeup to help physicians diagnose and treat a host or rare conditions. The information also provides families with a clear diagnosis, which helps reduce the stress and uncertainty that often accompanies undiagnosed diseases and disorders.

In 2019, the Center established the CSNK2A1 Foundation Research Program to accelerate progress toward a treatment and/or a cure for OCNDS. Led by its scientific director, Matt Huentelman, Ph.D., and medical director, Vinodh Narayanan, M.D., the Center’s collaboration with CSNK2A1 Foundation seeks to develop experiments in a controlled environment, like a test tube or petri dish, that mimic OCNDS. This gives researchers a tool to better understand the progression of OCNDS at the cellular level, as well as guide the development of potential therapies.

Your donation dollars at work.

We have assembled a team of scientists spanning the globe, many of whom worked on CSNK2A1 before it was associated with OCNDS. They are sharing data, collaborating, publishing together and meeting once a quarter at our Scientific Roundtable to accelerate the path to treatment. There is potential for treatment that removes the effect of the genetic mutations on CSNK2A1. We partnered with Unravel Biosciences, a state-ofthe-art Biotech company formed by two former Wyss Institute at Harvard University scientists. Using proprietary AI, they identified several FDA-approved compounds which they are testing on preclinical models such as frogs and mice in preparation for clinical trial readiness. This research has made it abundantly clear that a treatment is possible. In addition, until recently, we have run on the blood, sweat, and tears of our volunteers. With our donors' support, we hired a Science Program Director who is responsible for further developing and cultivating the Foundation’s research program.