Abstract

Human Liver Stem Cell-derived Nano-EVs – A Broad Platform Technology for Cancer and Regeneration

Daniel Gau, Head of Business Development and COO, Unicyte AG

Extracellular vesicles and exosomes (EVs), and in particular when derived from stem cells are emerging as a new therapeutic modality in regenerative medicine. Stem cell derived EVs are of high abundance, are not self-replicating, are not immunogenic, own a tissue specific targeting capability and thus address hurdles of stem cells for translation into indications with large patient impact. With more than 10 years research in the field, Unicyte has developed a strong therapeutic pipeline in fibrotic disorders and oncology, using it’s proprietary Human Liver Stem Cell and derived nano-EVs (HLSC-nEVs). With their natural targeting capability to liver tissue and to tumor cells, HLSC-nEVs have been investigated in Hepatocellular carcinoma (HCC) the most common cause of death in people with cirrhosis. In pre-clinical studies, HLSC-nEVs decrease the growth and survival rates of tumor liver cells, while at the same time protecting normal hepatocytes. In addition, HLSC-nEVs have demonstrated strong liver regeneration capabilities in various models of acute and chronic liver injury, ex-vivo scaffold re-cellularization and more recently on normothermic transplantation models. These results suggest that HLSC-nEV address the underlying liver disease and inhibit tumor growth in hepato-cellular carcinoma via a multi-factorial mechanism of action. Unicyte is currently preparing clinical transition of its HLSC-nEVs programs in various indications.