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RMX-1001 is an orally bioavailable small molecule designed to modulate the RNA processing of the **MYC** oncogene. Developed by Remix Therapeutics using their proprietary REMIX platform, the drug aims to selectively interfere with the splicing and processing of MYC mRNA, leading to a reduction in MYC protein levels. MYC is a central driver of cell proliferation and survival in numerous cancers but has historically been considered "undruggable" due to its lack of traditional small-molecule binding pockets. By targeting the mRNA processing stage, RMX-1001 effectively lowers MYC expression to inhibit tumor growth. It is currently being evaluated in Phase 1 clinical trials for the treatment of advanced solid tumors and hematologic malignancies.
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