Our approach is based on an elegant idea: a shielded transplant that enables cancer cells to 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, such as an ADC or CAR-T, 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.
Our clinical data has demonstrated that our approach has the potential to shield patients’ healthy cells and enable targeted therapies post-transplant. All patients treated with Mylotarg have demonstrated durable hematologic protection from deep cytopenias through repeat doses.