We believe our approach shows significant promise when potential targets fulfill three important criteria:
These targets are expressed on cancer cells
These targets are expressed on cells of hematopoietic lineage
There is evidence these protein targets are biologically dispensable
CD123, CLL-1, and EMR2 are targets expressed strongly in various myeloid blood cancers including AML. These targets are expressed both in bulk AML cells as well as leukemic stem cells. Our preclinical data demonstrates our ability to genetically engineer HSCs in human cells to remove expression of these targets with good efficiency. As such, we continue to research CD123, CLL-1, and EMR2 as potential target candidates for our shielded transplants and CAR-T therapies.
Novel CLL-1-directed CAR-T cells Mediate Potent Antigen-specific Cytolytic Activity in
Mouse Models of Acute Myeloid Leukemia
GUMM: A Purpose-Built Computational Workflow for Single Cell Genotyping of Gene Editing Experiments
A Single Cell DNA Sequencing Resource and Computational Approach to Quantify CRISPR-Cas9 Gene Editing Allelism
Gene Editing and Immunotherapeutic Targeting of ADGRE2/EMR2 to Enable Combinatorial Targeting in Acute Myeloid Leukemia (AML)
