Drug intelligence / Profile preview

GMR CAR-T cells

Development stage
Unknown
Lead developer
Shinshu University
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intravenous
01

Overview

GMR CAR-T cells are an investigational chimeric antigen receptor (CAR) T-cell therapy targeting CD116, also known as the granulocyte-macrophage colony-stimulating factor (GM-CSF) receptor alpha chain. Developed by researchers at Shinshu University, these cells are engineered using the non-viral piggyBac transposon system. The CAR construct typically incorporates a mutated GM-CSF ligand (e.g., E21K or E21R mutations) as the antigen recognition site to enhance anti-tumor activity and persistence, along with a CD28 costimulatory domain and CD3ζ signaling chain. The therapy is primarily being evaluated for the treatment of relapsed or refractory CD116-positive acute myeloid leukemia (AML) and juvenile myelomonocytic leukemia (JMML), where CD116 is frequently overexpressed.

Other names
CD116 CAR-T cellsCD-116 CAR-T cellsCD 116 CAR-T cellsGM-CSF receptor-targeted CAR-T cellspiggyBac-modified GMR CAR-T cells
02

Targets

CSF2RA (Granulocyte-macrophage colony-stimulating factor receptor subunit alpha)CSF2RB (Cytokine receptor common subunit beta)

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