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CT-01 is a preclinical drug candidate developed by Captor Therapeutics for the treatment of hepatocellular carcinoma (HCC), a form of liver cancer with significant unmet medical need. The drug is based on targeted protein degradation (TPD) technology and functions as a degradable molecular glue. Its mechanism involves inducing the degradation of key proteins implicated in tumorigenesis, specifically Eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1), Sal-like protein 4 (SALL4), and another undisclosed neo-substrate. Preclinical studies have demonstrated that oral administration of CT-01 compounds leads to complete tumor regression in mouse models of HCC without observed treatment-related toxicity. The unique degradation profile supports its competitive potential as an innovative therapy for HCC[1][5][7].
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