Deep conditioning using CRISPR-edited T cells  

Acute myeloid leukaemia (AML) is a cancer of white blood cells which is treated with chemotherapy followed by bone marrow transplant from a matched donor. If AML persists after these treatments, the outlook is very poor, with most children dying from the disease.

We have developed genome engineering techniques to collect and modify healthy donor immune cells (T cells) so they don't need to be matched to a specific patient, but are armed against AML and can help clear out a patient's bone marrow. This form of 'deep conditioning' uses multiple chimeric antigen receptors to eliminate any AML cells remaining after chemotherapy, and this will then be followed by a second transplant to repopulate the blood and immune system. 

A similar approach has shown promise against other forms of leukaemia and we believe early application in children with AML who are at the highest risk is now possible.