We are now working to bring our approach and the possibility of a cure to patients with high-risk Acute Myeloid Leukemia (AML), a disease that carries a poor prognosis even with aggressive therapies including Hematopoietic Cell Transplant (HCT), the current standard of care that has seen no significant change in decades.
Robust preclinical data supports the promise of our novel approach. Our engineered HSCs retain healthy stem cell function as demonstrated by the production of downstream progenitor cells with normal function. Furthermore, in preclinical models, transplantation with our engineered HSCs enables the successful clearing of AML cells with targeted therapies while preserving the normal blood cells.