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TTX-siPDL1 is an investigational small interfering RNA (siRNA) therapeutic developed by TransCode Therapeutics. It is designed to target and silence the expression of programmed death-ligand 1 (PD-L1), a key immune checkpoint protein that tumors use to evade immune detection. Unlike traditional antibody-based checkpoint inhibitors that block PD-L1 function, TTX-siPDL1 prevents the synthesis of PD-L1 at the mRNA level through RNA interference, leading to degradation or translational repression of PD-L1 mRNA and thus abolishing its expression on tumor cells. This approach aims to enhance T cell-mediated anti-tumor immunity and has shown robust preclinical efficacy in animal models of pancreatic cancer, especially when combined with gemcitabine chemotherapy. The drug is currently in preclinical development for pancreatic cancer and other solid tumors[1][4][5][7].
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