To drug genetically validated targets in novel ways, SyntheX's ToRFLEx platform engineers cells to survive based on a compound's specific mechanism of action.
What is the problem?
Over 85% of human proteins remain pharmacologically inaccessible. Binding-based discovery approaches can identify molecules that physically interact with a target in vitro, but they cannot confirm functional modulation of the protein in cells. This is particularly crucial for more complex drug mechanisms such as protein-protein interaction disruption or target degradation.
What is their solution?
Using an oncogene addiction and synthetic-lethality based target selection approach in Oncology, SyntheX designs screens to recapitulate genetic perturbations using novel pharmacology. The ToRFLEx platform relies on careful genetic engineering to enable precise selection-based drug discovery where cells only survive if a functional compound is present. This allows screening vast libraries of molecules to identify rare and potent compounds with specific a mechanism of action.