Regenerative Medicine

Regenerative medicine—the use of stem cells to repair or replace tissue or organ function lost due to disease, damage or age—holds tremendous potential in both rare and common diseases. We can use gene editing with the goal of enhancing these engineered cell therapies to treat diseases like diabetes.

To expand the potential of regenerative medicine, we are pursuing gene-editing approaches to allow allogeneic use of stem cell-derived therapies by enabling immune evasion with the goal of improving existing cell function and directing cell fate. Our first effort in this field focuses on developing a gene-edited beta-cell replacement product for the treatment of diabetes. We aim to use our gene-editing technology to protect transplanted cells from the patient’s immune system and improve the function of the transplanted cells by ex vivo editing of specific genes within the stem cell line used to produce the pancreatic beta-cell precursors.