MGTA-117 is a humanized monoclonal antibody that targets CD117. It carries an amanitin payload and is used to synthesize antibody-drug conjugates (ADC). MGTA-117 demonstrates potent antitumor activity, enhancing survival in three acute myeloid leukemia (AML) xenograft models [Kasumi-1, AML PDX 1, and AML PDX 2]. It is applicable for AML, myelodysplastic syndromes with excess blasts (MDS-EB), and preconditioning for hematopoietic stem cell transplantation (HSCT) in gene therapy.