hLEC™ (human liver engrafting cells)
The Therapeutic Potential of hLEC Cells
Our hLEC product candidate is enriched for progenitor cells, is directly transplantable and exhibits key characteristics that makes it attractive as a potential cell-based therapeutic, including demonstrated long-term engraftment in vivo. Preclinical studies demonstrate that hLEC cells exhibit hepatocyte function both in vitro and when transplanted in immunodeficient animals with a metabolic defect (FAH knockout mouse). These studies show that when engrafted, hLEC cells produce important proteins needed for proper metabolic function, which are missing or deficient in liver disease. Our hLEC cells are also distinguished by more consistent biochemical and metabolic activity compared to “standard” hepatocyte preparations. Additionally, hLEC cells can be derived from livers not otherwise transplantable and, in this way, would alleviate donor shortage issues and competition for donor organs from the organ transplant pool.
hLEC Assay for Drug Discovery
In addition to its therapeutic potential, we are currently evaluating an hLEC assay for toxicology screening of novel drug candidates. Liver toxicity continues to be one of the primary causes of clinical trial failures and market withdrawals of drug products. Our hLEC cells have been shown to exhibit metabolic and detoxification enzyme activities and could have significant utility in testing new drug candidates to detect adverse drug reactions before the start of clinical trials.
Patents
We hold a portfolio of issued or allowed US and foreign patents covering the methods of identification, isolation and use of hLEC cells, as well as the composition of the cells themselves, and are globally prosecuting these patent families in multiple jurisdictions throughout the world.