Protect the healthy cells,
expose the cancer

Our approach is based on an elegant idea: make the Hematopoietic Stem Cells (HSCs) resistant to cancer-directed therapies so that healthy blood cells are protected, but cancer cells can be directly targeted for destruction, potentially delaying or avoiding relapse post-transplant.

Healthy donor HSCs are first edited to remove a marker on the outside of the cells so that they can no longer be recognized by targeted therapies. After the transplant of the modified healthy donor cells, the patient may now be able to receive a targeted therapy to kill any remaining cancer cells and to decrease the chance that the cancer can come back because the healthy cells are now “invisible” to the targeted therapy.

This approach exhibited good activity and a favorable safety profile in our preclinical studies: when combined with our modified HSCs, targeted therapies eradicate the cancer cells while the normal healthy blood cells continue to thrive and function as normal.