Preclinical efficacy and safety evaluation of human embryonic stem cell-derived functional hepatocytes for a clinical trial in acute liver failure

A novel multistage, GMP-compliant process was developed for the effective and reproducible differentiation of hESCs into hepatocytes. The resulting HPLCs demonstrated robust hepatocyte functions, therapeutic efficacy in an ALF animal model, and favorable biosafety profiles. These findings support the clinical translation of HPLCs, with an ongoing Phase I clinical trial designed to evaluate their safety and feasibility in patients with ALF and ACLF.