The data reported in this pre-clinical study showed that transplantation of hpSC-derived hepatocytes cells (HLCs) elicits a significant decrease and long-term stabilization of bilirubin levels in the serum of Gunn rats, a well-established animal model of Crigler-Najjar syndrome type 1 (CN1), an inborn error of bilirubin metabolism. Moreover, at four months following treatment no adverse safety signals were detected.
CN1 is a rare inherited liver-based metabolic disorder in which the host liver lacks one hepatic enzyme, UGT1A1, essential for the conjugation and excretion of the toxin bilirubin. This pre-clinical study provides important supportive evidence of the potential safety and efficacy of HLCs which could constitute a reliable source for large numbers of transplantable cells. In the long-term, experience with HLC transplantation for CN1 could potentially be used to develop therapeutic strategies for more common inherited liver diseases.
"Being awarded such a prestigious honor is a testament to the quality of our scientific team at ISCO The data presented at this conference demonstrates the unique nature of our HLCs and their potential efficacy in a clinical setting." said Ruslan Semechkin, Ph.D., Chief Scientific Officer of ISCO.