Stem Cell Engineering

Creating immune-evasive cells for allogeneic cell replacement therapies

Because type 1 diabetes is an autoimmune disease that develops when the body’s immune system attacks and destroys the insulin-producing cells in the pancreas, any implanted cell replacement therapies must evade the immune system. Also, like donor organs, any implantable human cell product will express its own unique immune ‘signature,’ and thus patients will not be a match for that signature. Approaches to address this allogeneic mismatch can be physical (PEC-Encap) or pharmacological (immunosuppressive therapy with PEC-Direct). Alternatively, we believe it may be possible to genetically engineer the stem cells such that the cells made from them, such as PEC-01 cells, evade the immune system (PEC-QT).

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.

See Pipeline

ViaCyte and CRISPR have produced an immune-evasive pluripotent stem cell line.

“We believe the combination of regenerative medicine and gene editing has the potential to offer durable, curative therapies to patients in many different diseases, including common chronic disorders like insulin-requiring diabetes. ViaCyte is a pioneer in the regenerative medicine field, and has built a compelling clinical program, robust manufacturing capabilities, and assembled a strong intellectual property position,” commented Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. ”