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NRT-NMJ_001 is a preclinical-stage small interfering RNA (siRNA) therapeutic being developed by NEORNAT for the treatment of hepatocellular carcinoma (HCC). The drug's mechanism of action involves the siRNA-mediated silencing of a specific, overexpressed DNA replication enzyme that acts as an oncogene. By targeting this enzyme, NRT-NMJ_001 is designed to suppress the expression of DNA methyltransferases (DNMTs), thereby reversing aberrant DNA methylation patterns and restoring the expression of silenced tumor suppressor genes. Cell-level efficacy has been confirmed in preclinical studies, demonstrating its potential to inhibit tumor growth through epigenetic modulation.
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