CA3180738A1 - Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems - Google Patents

Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems Download PDF

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Publication number
CA3180738A1
CA3180738A1 CA3180738A CA3180738A CA3180738A1 CA 3180738 A1 CA3180738 A1 CA 3180738A1 CA 3180738 A CA3180738 A CA 3180738A CA 3180738 A CA3180738 A CA 3180738A CA 3180738 A1 CA3180738 A1 CA 3180738A1
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cell
hematopoietic stem
lineage
genetically engineered
grna
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CA3180738A
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English (en)
French (fr)
Inventor
Siddhartha MUKHERJEE
Florence Borot
Abdullah Mahmood Ali
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Columbia University in the City of New York
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0662—Stem cells
    • C12N5/0665—Blood-borne mesenchymal stem cells, e.g. from umbilical cord blood
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/15—Natural-killer [NK] cells; Natural-killer T [NKT] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
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    • A61K40/421—Immunoglobulin superfamily
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
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    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/4224—Molecules with a "CD" designation not provided for elsewhere
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the route of administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/46—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the cancer treated
    • A61K2239/48—Blood cells, e.g. leukemia or lymphoma
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
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CA3180738A 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems Pending CA3180738A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063033966P 2020-06-03 2020-06-03
US202063033970P 2020-06-03 2020-06-03
US63/033,970 2020-06-03
US63/033,966 2020-06-03
US202163183791P 2021-05-04 2021-05-04
US63/183,791 2021-05-04
PCT/US2021/035691 WO2021247856A2 (en) 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems

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Publication Number Publication Date
CA3180738A1 true CA3180738A1 (en) 2021-12-09

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CA3180738A Pending CA3180738A1 (en) 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems

Country Status (9)

Country Link
US (1) US20240041923A1 (https=)
EP (1) EP4161535A4 (https=)
JP (1) JP2023528415A (https=)
KR (1) KR20230029632A (https=)
CN (1) CN116157135A (https=)
AU (1) AU2021285907A1 (https=)
CA (1) CA3180738A1 (https=)
MX (1) MX2022015237A (https=)
WO (1) WO2021247856A2 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021081244A1 (en) * 2019-10-22 2021-04-29 Fred Hutchinson Cancer Research Center Base editor-mediated cd33 reduction to selectively protect therapeutic cells
CA3232593A1 (en) * 2021-09-14 2023-03-23 Vor Biopharma Inc. Compositions and methods for multiplex base editing in hematopoietic cells
CA3236152A1 (en) * 2021-11-09 2023-05-19 Vor Biopharma Inc. Compositions and methods for erm2 modification
CN120265314A (zh) * 2022-09-28 2025-07-04 瑞泽恩制药公司 抗体抗性修饰受体以增强基于细胞的疗法
CN116850210B (zh) * 2023-01-17 2024-05-31 武汉大学 用于治疗急性髓系白血病的成分和方法
WO2025030010A1 (en) * 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108290939B (zh) * 2015-10-16 2023-01-13 纽约市哥伦比亚大学理事会 用于抑制谱系特异性抗原的组合物和方法
JP2020523365A (ja) * 2017-06-14 2020-08-06 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを改変する方法
US11718659B2 (en) * 2017-08-28 2023-08-08 The Trustees Of Columbia University In The City Of New York CD33 exon 2 deficient donor stem cells for use with CD33 targeting agents
CA3110837A1 (en) * 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
WO2021081244A1 (en) * 2019-10-22 2021-04-29 Fred Hutchinson Cancer Research Center Base editor-mediated cd33 reduction to selectively protect therapeutic cells

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Publication number Publication date
EP4161535A4 (en) 2024-07-17
JP2023528415A (ja) 2023-07-04
KR20230029632A (ko) 2023-03-03
WO2021247856A2 (en) 2021-12-09
MX2022015237A (es) 2023-02-09
US20240041923A1 (en) 2024-02-08
CN116157135A (zh) 2023-05-23
AU2021285907A1 (en) 2023-02-02
EP4161535A2 (en) 2023-04-12
WO2021247856A3 (en) 2022-01-13

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