EFFECTIVE TARGETING OF PRIMARY HUMAN LEUKEMIA USING ANTI-CD123 CHIMERIC ANTIGEN RECEPTOR ENGINEERED T CELLS
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Abstract
The invention provides compositions and methods for treating leukemia, for example, acute myeloid leukemia (AML) and B-cell acute lymphoid leukemia (B-ALL). The invention also relates to at least one chimeric antigen receptor (CAR) specific to CD123, vectors comprising the same, and recombinant T cells comprising the CD123 CAR. The invention also includes methods of administering a genetically modified T cell expressing a CAR that comprises a CD123 binding domain. The invention also includes methods of bone marrow ablation for use in treatments necessitating bone marrow reconditioning or transplant.
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Citations
163 Claims
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1-102. -102. (canceled)
- 103. A method of eradicating at least a portion of existing bone marrow in a subject, the method comprising administering to the subject an effective amount of cells expressing a chimeric antigen receptor (CAR) molecule comprising an anti-CD123 binding domain, a transmembrane domain, and an intracellular signaling domain, thereby eradicating at least a portion of existing bone marrow in a subject.
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104-114. -114. (canceled)
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115. A method of treating a subject having cancer, the method comprising
administering to the subject an amount of cells expressing a chimeric antigen receptor (CAR) molecule effective to eradicate at least a portion of existing bone marrow in a subject, wherein said CAR molecule comprises an anti-CD123 binding domain, a transmembrane domain, and an intracellular signaling domain, thereby treating the subject having cancer.
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116. A method of treating a subject having a disease, disorder or condition that is treatable with a bone marrow transplant or bone marrow reconditioning, the method comprising
administering to the subject an amount of cells expressing a chimeric antigen receptor (CAR) molecule effective to eradicate at least a portion of existing bone marrow in a subject, wherein said CAR molecule comprises an anti-CD123 binding domain, a transmembrane domain, and an intracellular signaling domain, thereby treating the subject having a disease, disorder or condition that is treatable with a bone marrow transplant or bone marrow reconditioning.
Specification