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MYC-targeted rSM is an investigational RNA-targeted small molecule (rSM) being developed by Arrakis Therapeutics, potentially in collaboration with Roche. The program focuses on directly targeting the mRNA transcript of the MYC proto-oncogene, a high-value but historically 'undruggable' transcription factor that is frequently overexpressed in a wide range of human cancers. Unlike traditional small molecules that attempt to bind the MYC protein, which lacks a well-defined binding pocket, Arrakis's rSM approach identifies and binds to specific tertiary structures within the MYC mRNA. This binding is designed to inhibit MYC expression by interfering with protein translation or by promoting the degradation of the mRNA transcript, thereby reducing the levels of the oncogenic MYC protein and inhibiting tumor growth.
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