is pioneering the discovery and development of translation regulators for cancer and other serious diseases. eFFECTOR’s translation regulators are a new class of small molecules acting at protein targets that regulate translational control of gene expression.
For a small subset of proteins that drive key biological processes, post-transcriptional events (including translation initiation and mRNA stability) are more important drivers of expression levels than transcription rate. 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. Continued expression of these translationally dysregulated proteins becomes much more important to disease cells than healthy cells, because the disease cells rely on those proteins for continued survival and proliferation. This provides a therapeutic window when translational control is restored. Because many functionally related genes are up-regulated in a coordinated fashion in disease, restoring control to the translational landscape may down-regulate multiple disease drivers simultaneously.
eFFECTOR’s most advanced program is eFT508, an inhibitor of MNK 1 and MNK 2 kinases, being developed in oncology. MNK 1, 2 kinases integrate signals from multiple oncogenic pathways to regulate multiple disease drivers. eFFECTOR has demonstrated significant efficacy in multiple human tumor models, including solid tumors and lymphoma. We expect to file an IND for eFT508 in 2015.
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