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BSB-1001 is an investigational T cell receptor (TCR)-engineered T cell therapy developed by BlueSphere Bio for the treatment of hematologic malignancies. The therapeutic consists of donor-derived CD8+ T cells transduced with a lentiviral vector encoding a high-affinity TCR specific for the minor histocompatibility antigen-1 (HA-1). HA-1 is a blood-restricted antigen expressed predominantly on hematopoietic cells, including leukemia cells, but not on healthy non-hematopoietic tissues. This restricted expression profile is designed to mediate a potent graft-versus-leukemia (GvL) effect while minimizing the risk of graft-versus-host disease (GvHD). The product also incorporates the RQR8 safety tag, which allows for the in vivo elimination of BSB-1001 cells using rituximab if severe toxicity occurs. In the 'SOC + BSB-1001' regimen, the cell therapy is administered in combination with standard-of-care (SOC) T cell-depleted allogeneic hematopoietic stem cell transplantation (alloHSCT) to prevent relapse in patients with high-risk or relapsed/refractory acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic syndrome (MDS).
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