Preferred Label : Anti-CD117/Amanitin Antibody-drug Conjugate MGTA-117;
NCIt synonyms : ADC MGTA-117; Anti-c-Kit/Amanitin ADC MGTA-117; Anti-CD117 ADC MGTA-117;
NCIt definition : An antibody-drug conjugate (ADC) consisting of a human monoclonal antibody directed
against CD117 (tyrosine-protein kinase KIT; c-Kit; mast/stem cell growth factor receptor;
SCFR), conjugated, via a cleavable linker, to the cytotoxic RNA polymerase II inhibitor
amanitin, with potential antineoplastic activity and that can potentially be used
as a conditioning agent to selectively deplete hematopoietic stem cells (HSCs) prior
to allogeneic HSC transplants or other treatments for which HSCs need to be depleted.
Upon administration of the anti-CD117/amanitin ADC MGTA-117, the antibody moiety targets
and binds to the cell surface antigen CD117. Upon internalization and cleavage, the
cytotoxic amanitin is released, binds to and inhibits RNA polymerase II. This prevents
RNA synthesis and induces apoptosis of CD117-overexpressing HSCs and leukemic blasts.
CD117, a transmembrane protein and receptor tyrosine kinase, is highly expressed on
HSCs, progenitor cells and on leukemia cells. MGTA-117 is engineered to have a short
half-life.;
Molecule name : MGTA 117; MGTA-117;
NCI Metathesaurus CUI : CL1778894;
Origin ID : C186516;
UMLS CUI : C5667067;
Semantic type(s)
- concept_is_in_subset
- has_target
