Concise Review: Manufacturing of Pancreatic Endoderm Cells for Clinical Trials in Type 1 Diabetes

Abstract

Abstract The cellular component of ViaCyte's VC-01 combination product for type 1 diabetes, pancreatic endoderm cells (PEC-01) derived from CyT49 human embryonic stem cells, matures after transplantation and functions to regulate blood glucose in rodent models. The aims in manufacturing PEC-01 at scale are to generate a consistent and robust transplantable population that functions reliably and safely in vivo. ViaCyte has integrated multiple bioprocessing strategies to enable a tightly controlled PEC-01 manufacturing process for clinical entry. Significance This review summarizes the manufacturing process for the first human embryonic stem cell-based candidate treatment for type 1 diabetes to enter clinical testing. It presents the key strategic principles used to enable scaled production of pancreatic endoderm and will have widespread interest in academia and biopharma.