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ACK2 is a monoclonal antibody that targets and blocks the c-Kit receptor (CD117), a 145 kDa cytokine receptor found on hematopoietic stem cells (HSCs). By antagonizing the binding of its ligand, stem cell factor (SCF), ACK2 inhibits the survival and proliferation of HSCs. In preclinical research, it is utilized as a non-genotoxic conditioning agent to deplete host HSCs, thereby facilitating the engraftment of donor cells during hematopoietic stem cell transplantation. It has been specifically investigated for treating inherited hematologic disorders such as X-linked chronic granulomatous disease (X-CGD) and for in utero hematopoietic cell transplantation. While ACK2 alone shows limited efficacy in some settings, its potency is significantly enhanced when combined with low-dose irradiation.
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