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cd117-saporin is an experimental antibody-drug conjugate that couples an anti-CD117 (c-Kit) monoclonal antibody to the ribosome-inactivating toxin saporin, typically via a streptavidin–biotin linkage, to selectively ablate hematopoietic stem and progenitor cells without genotoxic chemotherapy or irradiation.[2][7][11] By binding CD117 on hematopoietic stem cells, internalizing, and releasing saporin intracellularly, the conjugate halts protein synthesis and induces cell death, enabling efficient donor hematopoietic cell engraftment and durable hematopoietic chimerism in mouse models while largely preserving downstream immune effector cells and overall immunity.[2][7] This non-myeloablative conditioning approach has been developed primarily in academic settings (e.g., Stanford/Harvard collaborations) as a platform strategy to improve the safety of hematopoietic stem cell transplantation and potentially support gene therapy and tolerance protocols for a range of malignant and non-malignant hematologic and immune diseases.[2][6][7]
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