eFFECTOR Therapeutics is a clinical-stage biopharmaceutical company focused on pioneering the development of a new class of oncology drugs known as selective translation regulator inhibitors (STRIs). Translation is the process in cells whereby the synthesis of proteins is directed by information contained in genetic sequences. Our targets lie at the bottom of two of the most frequently mutated signaling pathways in cancer—the RAS and PI3K pathways—and are responsible for the translation of select messenger RNA (mRNA) into proteins, which are frequent culprits in key disease-driving processes. Each of our product candidates is designed to act on a single protein that regulates, in a coordinated manner, the expression of multiple functionally related proteins that together drive tumor cell proliferation and survival, cell cycle progression and important biological processes such as tumor immune evasion.

In cancer, the tightly controlled translation of certain mRNA frequently becomes upregulated, leading to production of oncoproteins that cause uncontrolled growth, immune evasion and metastasis. We believe that our therapeutic approach can inhibit the production of key disease-driving proteins, which tumors have hijacked for their benefit and are highly sensitive to their inhibition, all while preserving normal cell function.

Utilizing our proprietary selective translation regulation technology platform, we have developed an understanding of genes that are translationally upregulated in multiple tumor types. This has enabled us to identify specific points of therapeutic intervention that may have a meaningful clinical effect. We believe our in-depth understanding of translation regulation biology combined with our sophisticated structure-based design approach to medicinal chemistry has given us a key advantage in pioneering the emerging field of translation regulation therapeutics and creating significant barriers to entry.

Certain RNA viruses, including SARS-CoV-2, the causative agent of COVID-19, hijack the host cell translational machinery to drive viral replication. Because of this, we are planning to test one of our STRIs, zotatifin, in patients with mild-to-moderate COVID 19.

eFFECTOR was founded on pioneering research in the laboratories of Drs. Davide Ruggero and Kevan Shokat and subsequently licensed proprietary applications of translational profiling technology from the University of California, San Francisco. The company’s scientific founders and management team comprise industry veterans who have played important roles in the discovery and development of marketed small molecule drugs, monoclonal antibody therapeutics and cellular immunotherapies in oncology and other disease areas.