CD19 gene-edited hematopoietic stem cells

CD19 gene-edited hematopoietic stem cells are an investigational cell and gene therapy designed to prevent prolonged B-cell aplasia, a common adverse effect of CD19-targeted Chimeric Antigen Receptor T-cell (CAR-T) therapies used for B-cell malignancies. This therapy involves modifying hematopoietic stem cells (HSCs) using advanced gene editing techniques, such as CRISPR-Cas9 and prime editing. The goal is to alter the CD19 gene in these HSCs so that the B cells they produce lack the FMC63-CAR binding motif on the CD19 antigen, rendering them resistant to depletion by FMC63-CD19-CAR-T cells. These engineered B cells are intended to retain normal functionality and immune response capabilities, thereby supporting immune reconstitution and improving long-term patient outcomes by balancing therapeutic efficacy with immune system preservation.