By protecting healthy blood cells, our approach to Hematopoietic Stem Cell Transplant (HSCT) expands the anti-leukemia armamentarium for patients with AML and other blood cancers who have a poor prognosis with a high risk of relapse.
Our proprietary approach genetically modifies healthy donor HSCs to remove select cell surface targets that are shared between healthy blood cells and cancer cells. This would enable targeted therapies to destroy cancer cells selectively while preserving healthy blood cells. Protection of healthy blood cells could expand the use of different therapeutic approaches following transplant that have not been possible previously. By combining our engineered HSCs with targeted leukemia therapies—including Antibody Drug Conjugates (ADCs) and Chimeric Antigen Receptor T (CAR-T) cells—we hope to advance the standard of care for AML and other blood cancers.
Robust preclinical data supports the promise of this novel approach. This data demonstrates that our engineered HSCs retain healthy cell function as demonstrated by the production of downstream progenitor cells with normal function. Furthermore, in preclinical models, transplant with our engineered HSCs enables the successful clearing of AML cells with targeted therapies while preserving the normal blood cells.