eFFECTOR Therapeutics is pioneering the discovery and development of translation regulators as a new class of small molecule therapeutics that control gene expression for the treatment of cancer and other serious diseases.

Control of the translation of mRNA into protein is critical in order to maintain the precisely orchestrated processes carried out by proteins within a cell. In certain diseases, especially cancer, the tightly controlled translation of specific proteins becomes dysregulated, leading to inappropriate and often higher levels of production of these proteins than occurs in healthy tissue.

Rather than targeting disease-triggering proteins, such as mutated oncogenes, eFFECTOR’s approach is to treat disease by selectively controlling the production of proteins that carry out the consequences of the trigger. It is these downstream proteins that actually result in harm and disease progression.  The result is an entirely novel class of drugs offering key benefits to patients, including oral delivery and the potential for more durable benefit due to a lower likelihood of developing resistance.

eFFECTOR was formed based on research breakthroughs made in the laboratories of University of California, San Francisco scientists Davide Ruggero, Ph.D., and Kevan Shokat, Ph.D. The key discovery of Drs. Ruggero and Shokat leveraged by eFFECTOR is that certain functionally-related sets of proteins, or regulons, that drive disease when upregulated, are translationally controlled. The scientists discovered that restoring translational control of those regulons can address disease pathology while preserving normal cell functions.  Because multiple oncogenic signaling pathways activate translation, translation regulators may also delay or prevent a common mechanism of drug resistance to targeted therapeutic intervention, namely activation of adjacent pathways.