In this project, we will refine protocols to enable the production of sufficient amount EPCs that are compliant with applicable current Good Manufacturing Practice (cGMP), characterize their composition and biological features, and evaluate robustly their therapeutic potencies for endothelial reconstitution in vivo. Eventually, we will formulate them as clinical-stage-appropriate products for further research and development. Deriving EPCs from ESCs and testing their efficacy in the same species, especially in baboons, will fundamentally impact embryonic stem cell therapy because 50% of clinical trails of cell therapeutics are conducted without an understanding of their mode of action, which has resulted in a significant economic loss. An objective and reproducible evaluation of therapeutic biologics is needed before further clinical application can be optimized. While many ESC-derived cells appear to be attractive options, standard and robust in vitro and in vivo tests in a suitable animal model can identify those that are the most amenable to therapeutic application.
