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.
Authors
- Menghan Xie
- Qi Ouyang (ORCID: https://orcid.org/0000-0003-1585-7368)
- Guangxiu Lu
- Ge Lin
- Baicun Zhou
- Yi Sun
- Caiyun Xu
- Yang Wang
- Yang Shi
- Xingxiang Duan
- Mei Wang
- Yan Zhao
- Juan Yu
Institutions
- Ministry of Education of the People's Republic of China (CN)
- Central South University (CN)
- Reproductive & Genetic Hospital CITIC-Xiangya (CN)
- National Engineering Research Center of Human Stem Cells (CN)
- Changsha University of Science and Technology (CN)
Publication Details
- Journal
- Stem Cell Research & Therapy
- Published
- 2026-03-23
- DOI
- https://doi.org/10.1186/s13287-026-04985-8
- Primary Topic
- Pluripotent Stem Cells Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00