A recent established target, folate receptor 1 (FOLR1), overexpressed in AML, was shown to have no impact on normal HSPCs in C/G positive pediatric acute megakaryoblastic leukemia, but it is expressed in various healthy tissues (kidney, intestine, lung, retina, placenta, and choroid plexus) and severe lung toxicity has already been demonstrated in studies with a T cell bispecific antibody against FOLR1 in nonhuman primates [40]. The gene discussed is FOLR1; the disease is acute megakaryoblastic leukemia.