KIT and severe combined immunodeficiency: Clinically, c-kit is the target of monoclonal antibody briquilimab, which has shown promise in treating patients with AML and myelodysplastic syndromes (MDS) in a Phase 1 clinical trial (NCT04429191), Fanconi anemia in a Phase 1/2 clinical trial (NCT04784052), severe combined immunodeficiency (SCID) in a Phase 1/2 clinical trial (NCT02963064), and sickle cell disease in a Phase 1/2 clinical trial (NCT05357482) with no treatment-related adverse events in 130 dosed subjects as of late September 2023 (23, 24).