Since its emergence, CRISPR technology has brought a tremendous leap forward in the biopharmaceutical industry. In collaboration with ViaCyte, CRISPR Therapeutics has developed two drug candidates, VCTX210 and VCTX211, both of which are gene-edited, allogeneic, stem cell-derived candidates for the treatment of type 1 diabetes. By transplanting pancreatic cells differentiated from stem cells, these therapies can restore insulin secretion in patients with type 1 diabetes, achieving the goal of functional cure.
These products use CRISPR/Cas9 to knock out the protein genes on the surface of stem cells that are targeted by T cells, so that the transplanted cells will not be attacked by the host’s immune system. At present, both products have entered clinical trials.