Preclinical in vitro and in vivo models have shown that NY-303 is highly potent and effective in mediating NK cell-redirected cytotoxicity against multiple HCC tumor cell lines [154] and in down-modulating GPC3 expression and inhibiting beta-catenin activation, resulting in a PD-1 checkpoint blockade-sensitive biomarker signature [111]. The gene discussed is PDCD1; the disease is neoplasm.