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 these targets as potential target candidates for our shielded transplants and CAR-T therapies.
Analyzing Guide RNA (gRNA) Impurity Discovery & Quantification
Purification of CAR+ T Cells Reveals Impact of Untransduced Cells in CAR-T Drug Product
Gene-Edited Hematopoietic Stem Cells to Enable Next-Generation CAR-T Cell Therapy for the Treatment of AML
Uncovering Molecular and Functional Dynamics of Human Hematopoietic Stem Cells during In Vitro Culture for Cell Therapy Manufacturing
Leveraging CRISPR/Cas9 and HDR to create an engineered CD33 CAR-T to target AML
CD33-Deleted Hematopoietic Cells (Trem-Cel) Are Protected from CD33xCD3 Bispecific Antibody Treatment and Produce Significantly Reduced Levels of Inflammatory Cytokines in Preclinical Studies
Multimodal Atlas of Paired Diagnosis and Relapse AML Samples Enables Novel Therapeutic Targeting of Surface Antigens
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
