MGTA-117

MGTA-117 is a novel antibody-drug conjugate (ADC) designed for targeted conditioning prior to hematopoietic stem cell transplantation (HSCT). It consists of a human monoclonal antibody directed against CD117 (c-Kit, tyrosine-protein kinase KIT), which is highly expressed on hematopoietic stem cells and certain tumor cells, including those in acute myeloid leukemia (AML) and myelodysplastic syndrome with excess blasts (MDS-EB). The antibody is conjugated to an amanitin payload, a potent inhibitor of RNA polymerase II. By binding to CD117-expressing cells, MGTA-117 delivers the cytotoxic amanitin specifically to these targets, leading to their depletion. This selective mechanism aims to clear space in the bone marrow for donor cell engraftment while minimizing off-target toxicity. The drug has shown high selectivity and efficacy in preclinical models and entered clinical trials as a single-agent conditioning regimen for patients with relapsed/refractory AML or MDS-EB undergoing HSCT[1][2][3][4][5].

Other names

anti-CD117/amanitin antibody-drug conjugate