hepatocyte-like cell therapy

Hepatocyte-like cell therapy, specifically the HAES (Human Embryonic Stem cell-derived HepAtocyte-like cells) program, is an allogeneic cell therapy developed by the National Center for Child Health and Development (NCCHD) in Japan. It consists of functional hepatocytes derived from human embryonic stem cells (hESCs) and is designed as a bridge treatment for infants with neonatal-onset urea cycle disorders (UCD) who are awaiting liver transplantation. The therapy is administered via intrahepatic transplantation through portal vein injection. Once engrafted, these cells provide the necessary urea cycle enzymes—such as ornithine transcarbamylase (OTC) and carbamoyl-phosphate synthase 1 (CPS1)—to metabolize ammonia, thereby preventing hyperammonemia and associated neurological damage during the critical neonatal period before a definitive liver transplant can be performed.

Other names

hepatocyte-like cell therapyhESC-derived hepatocyte-like cellshuman embryonic stem cell-derived hepatocyte-like cells