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UTR-cMYC is an investigational RNA-based therapeutic developed by UTR Therapeutics that targets the c-MYC (MYC) oncogene, a master regulator of cell growth and metabolism frequently dysregulated in cancer. The drug employs a novel 'RNA rewriting' modality, which involves engineering destabilized AU-rich elements (AREs) into the 3' untranslated region (UTR) of the target mRNA. This mechanism is designed to selectively destabilize c-MYC mRNA, leading to its rapid degradation and a subsequent reduction in c-MYC protein levels. By targeting the mRNA rather than the protein, UTR-cMYC aims to overcome the historical challenges of drugging the c-MYC transcription factor with small molecules. The program is currently in Phase 1 clinical development for multiple solid tumors, including triple-negative breast cancer (TNBC), pancreatic cancer, ovarian cancer, and colon cancer, with additional preclinical research in osteosarcoma.
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