Genetically-modified cells and method for producing same
AssigneesKissei Pharmaceutical Co., Ltd., Shinshu University
InventorsYozo Nakazawa, Kazuyuki Matsuda, Shigeru Nakano
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Abstract
Production of a chimeric antigen receptor (CAR) expressing cell having excellent target cytotoxicity. Provided is a genetically modified cell comprising, introduced thereinto, a polynucleotide encoding a chimeric antigen receptor (CAR) protein having a target binding domain that specifically binds to a human granulocyte-macrophage colony stimulating factor (GM-CSF) receptor, a transmembrane domain and an intracellular signaling domain.
Claims6
A method of treating juvenile myelomonocytic leukemia (JMML) or acute myelogenous leukemia (AML) in a human patient, the method comprising:
- intravenously administering a pharmaceutical composition comprising a pharmaceutically acceptable carrier and cells that are genetically modified to express a chimeric antigen receptor (CAR) protein,
- wherein the CAR protein comprises a target binding domain that specifically binds to a human granulocyte-macrophage colony stimulating factor (GM-CSF) receptor, a transmembrane domain and an intracellular signaling domain, and
- wherein the genetically modified cells exert cytotoxic activity against leukemia cells in the human patient.
Dependencies are inferred from the claim text.