Development of regenerative medicine therapies for liver failure

Period of Performance: 09/15/2017 - 03/31/2018


Phase 1 SBIR

Recipient Firm

Hepatx Corporation
Principal Investigator


Late-stage liver disease (LSLD) and is a contributing factor in more than 30,000 deaths per year in the US. Liver transplantation is the only disease-modifying treatment for LSLD, but this solution is impeded by a chronic shortage of available organs. HepaTx Corporation has licensed technology developed in the laboratory of Prof. Gary Peltz at Stanford University to develop a therapeutic strategy that will replace liver transplantation with stem cell-based liver regeneration. To this end, adipose-derived stem cells are isolated from discarded liposuction material, then they are differentiated in culture into induced hepatocytes (SF-Heps) to be infused into patients for liver regeneration. The planned first use of SF-Heps in humans will be in a phase I clinical trial for the treatment of acute liver failure. Transplantation of hepatocytes derived from human livers has already been demonstrated to be safe, however sourcing of suitable human cells impedes the widespread application of this treatment strategy. HepaTx?s technology can produce enough hepatocytes to regenerate an entire human liver from the amount of material obtained in a standard liposuction procedure. SF-Heps are produced under GMP compatible conditions, do not form tumors, and have been shown to reconstitute functional human liver tissue in a mouse model. The goal of this Phase 1 SBIR application is to confirm and further characterize SF-Hep function in vitro and demonstrate their clinical utility in vivo. More specifically, hepatocyte function of SF-Heps will be tested in vitro for similarity to published data on hepatocytes. SF-Heps will also be transplanted into mice using a model of acute liver failure. SF-Heps will be evaluated for impact on disease pathophysiology and engraftment. employed by the Peltz laboratory for testing SF-Hep transplantation. These studies will generate data that will inform additional translational activities in Phase 2, including the development of a robust protocol for manufacturing clinical-grade material and additional in vivo modeling of liver disease.