GMR CAR-T cells (E21K)

GMR CAR-T cells (E21K) is an experimental chimeric antigen receptor (CAR) T-cell therapy designed to target the granulocyte-macrophage colony-stimulating factor receptor (GMR), specifically the CD116/CD131 complex, which is highly expressed on myeloid leukemia cells. Developed by researchers at Shinshu University, this therapy utilizes a ligand-based CAR construct where the antigen-recognition domain is a mutated version of the human GM-CSF protein. The specific E21K mutation (glutamate-to-lysine substitution at residue 21) is incorporated to enhance anti-tumor efficacy and persistence compared to wild-type ligand-based CARs. The construct is delivered via the non-viral piggyBac transposon system and includes a G4S spacer, a CD28 costimulatory domain, and a CD3ζ signaling domain. It is primarily being investigated for the treatment of relapsed or refractory acute myeloid leukemia (AML) and juvenile myelomonocytic leukemia (JMML).

Other names

mutated GM-CSF-based CAR T-cellsCD116-targeted CAR-T cellsCD-116-targeted CAR-T cellsCD 116-targeted CAR-T cellsGMR CAR-T cells