CD33 base-edited hematopoietic stem and progenitor cells

CD33 base-edited hematopoietic stem and progenitor cells (HSPCs) represent an ex vivo cell and gene therapy approach designed to enable more intensive CD33-targeted treatment for acute myeloid leukemia (AML). By utilizing adenine base editing (ABE), a specific single nucleotide polymorphism is introduced into the CD33 gene of donor or patient-derived HSPCs. This modification alters the epitope targeted by CD33-directed agents — such as the antibody-drug conjugate gemtuzumab ozogamicin or CD33-targeted CAR T cells — rendering the resulting myeloid progeny resistant to treatment-induced depletion. Unlike gene knockout strategies, this epitope-shielding method preserves the surface expression and native function of CD33, potentially avoiding long-term biological consequences of antigen loss while preventing dose-limiting myelotoxicity. This platform is being developed by Cimeio Therapeutics in collaboration with the University of Basel.

Other names

CD33 base-edited HSPCsCD-33 base-edited HSPCsCD 33 base-edited HSPCsCD33-edited HSPCsCD-33-edited HSPCsCD 33-edited HSPCsepitope-shielded CD33 HSPCs