Using gene editing on the pluripotent stem cell starting material offers the potential to protect implanted cells from the patient’s immune system by ex vivo editing immune-modulatory genes in ViaCyte’s CyT49 stem cell line that is used to produce the pancreatic lineage cells. We believe the speed, specificity, and multiplexing efficiency of the CRISPR-Cas9 gene editing technology make it ideally suited to this task.
ViaCyte and CRISPR Therapeutics, a leading company in the gene-editing space, are collaborating to discover, develop, and commercialize an immune-evasive islet replacement treatment for diabetes, which we refer to as the PEC-QT program.
CRISPR Therapeutics has significant expertise in immune-evasive gene editing. The combination of ViaCyte’s stem cell capabilities and CRISPR’s gene editing capabilities has the potential to enable an islet replacement product that may deliver durable benefit to patients without triggering immune rejection.
An immune-evasive version of ViaCyte’s CyT49 cell line could also be used to pursue treatments for other human diseases that are caused by a loss or malfunction of a specific cell or tissue type.