KR20240043783A - 유전자 변형 세포의 제조 방법 - Google Patents

유전자 변형 세포의 제조 방법 Download PDF

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Publication number
KR20240043783A
KR20240043783A KR1020247007517A KR20247007517A KR20240043783A KR 20240043783 A KR20240043783 A KR 20240043783A KR 1020247007517 A KR1020247007517 A KR 1020247007517A KR 20247007517 A KR20247007517 A KR 20247007517A KR 20240043783 A KR20240043783 A KR 20240043783A
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South Korea
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cells
rna
cell
sequence
car
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English (en)
Korean (ko)
Inventor
셍칸 진
후안-카를로스 콜란테스
존 람부른
임마콜라타 포레카
토마소 셀미
Original Assignee
럿거스, 더 스테이트 유니버시티 오브 뉴 저지
호리존 디스커버리 리미티드
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Publication of KR20240043783A publication Critical patent/KR20240043783A/ko
Pending legal-status Critical Current

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • A61K40/421—Immunoglobulin superfamily
    • A61K40/4211—CD19 or B4
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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
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    • C12N15/09—Recombinant DNA-technology
    • 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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    • 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
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    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
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    • C12N5/0602—Vertebrate cells
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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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/10—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the structure of the chimeric antigen receptor [CAR]
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A—HUMAN NECESSITIES
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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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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  • Wood Science & Technology (AREA)
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  • Biophysics (AREA)
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  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
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  • Virology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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KR1020247007517A 2021-08-06 2022-08-05 유전자 변형 세포의 제조 방법 Pending KR20240043783A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163203996P 2021-08-06 2021-08-06
US63/203,996 2021-08-06
PCT/US2022/074625 WO2023015307A1 (en) 2021-08-06 2022-08-05 Method for producing genetically modified cells

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KR20240043783A true KR20240043783A (ko) 2024-04-03

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KR1020247007517A Pending KR20240043783A (ko) 2021-08-06 2022-08-05 유전자 변형 세포의 제조 방법

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US (1) US20250109413A1 (enExample)
EP (1) EP4380627A1 (enExample)
JP (1) JP2024534720A (enExample)
KR (1) KR20240043783A (enExample)
CN (1) CN118159301A (enExample)
AU (1) AU2022324118A1 (enExample)
CA (1) CA3227964A1 (enExample)
WO (1) WO2023015307A1 (enExample)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3322804B1 (en) 2015-07-15 2021-09-01 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
WO2025070822A1 (ja) * 2023-09-29 2025-04-03 味の素株式会社 Ｈｌａ遺伝子改変細胞
WO2025117745A1 (en) * 2023-12-01 2025-06-05 Caribou Biosciences, Inc. Methods and compositions for improving precision of endonuclease-mediated genome editing
WO2025179057A1 (en) * 2024-02-21 2025-08-28 The Regents Of The University Of California In vivo modification of cell genomes

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7519660B2 (ja) * 2016-06-14 2024-07-22 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 疾患を処置するための遺伝子改変された細胞、組織、および臓器
CA3151279A1 (en) * 2019-09-17 2021-03-25 Shengkan Jin Highly efficient dna base editors mediated by rna-aptamer recruitment for targeted genome modification and uses thereof

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US20250109413A1 (en) 2025-04-03
WO2023015307A1 (en) 2023-02-09
AU2022324118A1 (en) 2024-02-22
CA3227964A1 (en) 2023-02-09
CN118159301A (zh) 2024-06-07
EP4380627A1 (en) 2024-06-12

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