eFFECTOR Therapeutics

eFFECTOR Therapeutics is pioneering a new class of small molecule drugs that act by selectively regulating translation, also known as protein synthesis. The company unites breakthrough insights into the mechanisms of translational control with a proven approach to product invention. We expect this fusion to yield important new therapies for cancer and other serious diseases.
Translation was identified as a fundamental step in gene expression more than fifty years ago, but was only recently recognized as a control point regulating the levels of key proteins during important cellular events. In the healthy state, controlled translation enables a coordinated and usually transient response to growth stimuli and other cellular conditions. When translational control is lost, due to mutations or other disruptive conditions, certain pathways become inappropriately activated and disease frequently ensues. eFFECTOR is pursuing selective translation regulators to restore homeostasis to the translation landscape, thereby reversing the consequences of disregulation and offering treatment for a variety of serious conditions.
Cancer therapy is a particularly promising application of our technology. Multiple oncogenes activate translation, resulting in the up-regulation of several biological mechanisms important for cancer, including cell growth and division, cell motility and metastasis. eFFECTOR’s very name derives from this concept, translation being an effector function required to transform cause (oncogene mutation) into effect (pathway activation). Tumor cells that continue to evolve in the presence of continuously activated translation become dependent on, or “addicted to,” the disregulation. By inhibiting a critical dependency of multiple oncogenes, selective translation regulators offer the potential to treat a variety of tumor types and extend treatment benefit beyond what has been observed with inhibitors of single oncogenic drivers. eFFECTOR’s academic co-founders, Drs. Davide Ruggero and Kevan Shokat of UC San Francisco, are world leaders in mapping the connection between disregulated translation and cancer.
eFFECTOR’s approach to inventing new small molecule drugs blends the best practices of structure-based and fragment-based drug design. Binding sites on targets are experimentally interrogated with probe molecules to reveal key atomic interactions. These interactions are subsequently incorporated into novel scaffolds with high ligand efficiencies, providing a strong foundation for further optimization of biological and physicochemical properties. Drug candidates previously discovered by these methods have proven to have favorable clinical profiles. eFFECTOR’s drug discovery team is led by individuals who have made seminal contributions to this approach over the past 25 years.