CA3135198A1 - Generation of chimeric antigen receptor (car)-primary nk cells for cancer immunotherapy using a combination of cas9/rnp and aav viruses - Google Patents

Generation of chimeric antigen receptor (car)-primary nk cells for cancer immunotherapy using a combination of cas9/rnp and aav viruses Download PDF

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Publication number
CA3135198A1
CA3135198A1 CA3135198A CA3135198A CA3135198A1 CA 3135198 A1 CA3135198 A1 CA 3135198A1 CA 3135198 A CA3135198 A CA 3135198A CA 3135198 A CA3135198 A CA 3135198A CA 3135198 A1 CA3135198 A1 CA 3135198A1
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Prior art keywords
cell
cells
transgene
plasmid
aav
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Pending
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CA3135198A
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English (en)
French (fr)
Inventor
Dean Anthony LEE
Kathrin Christine Meyer
Meisam Naeimi KARAROUDI
Shibi LIKHITE
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Nationwide Childrens Hospital Inc
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Nationwide Childrens Hospital Inc
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Publication of CA3135198A1 publication Critical patent/CA3135198A1/en
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • 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
    • A61K40/428—Undefined tumor antigens, e.g. tumor lysate or antigens targeted by cells isolated from tumor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C—CHEMISTRY; METALLURGY
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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
    • C12N5/0646—Natural killers cells [NK], NKT 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K2121/00—Preparations for use in therapy
    • A—HUMAN NECESSITIES
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20—Cytokines; Chemokines
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    • C12N2501/2321—Interleukin-21 (IL-21)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified cells
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Oncology (AREA)
CA3135198A 2019-03-27 2020-03-27 Generation of chimeric antigen receptor (car)-primary nk cells for cancer immunotherapy using a combination of cas9/rnp and aav viruses Pending CA3135198A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962825007P 2019-03-27 2019-03-27
US62/825,007 2019-03-27
PCT/US2020/025454 WO2020198675A1 (en) 2019-03-27 2020-03-27 Generation of chimeric antigen receptor (car)-primary nk cells for cancer immunotherapy using a combination of cas9/rnp and aav viruses

Publications (1)

Publication Number Publication Date
CA3135198A1 true CA3135198A1 (en) 2020-10-01

Family

ID=72611833

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3135198A Pending CA3135198A1 (en) 2019-03-27 2020-03-27 Generation of chimeric antigen receptor (car)-primary nk cells for cancer immunotherapy using a combination of cas9/rnp and aav viruses

Country Status (14)

Country Link
US (1) US20220177917A1 (https=)
EP (1) EP3946432A4 (https=)
JP (2) JP7702878B2 (https=)
KR (1) KR20220017887A (https=)
CN (1) CN113891727A (https=)
AU (1) AU2020248097A1 (https=)
BR (1) BR112021019180A2 (https=)
CA (1) CA3135198A1 (https=)
CO (1) CO2021014353A2 (https=)
IL (1) IL286701A (https=)
MX (1) MX2021011782A (https=)
PH (1) PH12021552383A1 (https=)
SG (1) SG11202110613SA (https=)
WO (1) WO2020198675A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12473336B2 (en) 2018-02-21 2025-11-18 Board Of Regents, The University Of Texas System Methods for activation and expansion of natural killer cells and uses thereof
MX2023004811A (es) * 2020-10-26 2023-06-29 Res Institute At Nationwide Children´S Hospital Células nk del receptor de antígeno quimérico (car) y usos de las mismas.
WO2022236174A1 (en) * 2021-05-07 2022-11-10 Washington University In situ car-t therapies, vectors and methods therefor
US20240254445A1 (en) * 2021-06-01 2024-08-01 Vaxcell-Bio Composition containing feeder cell for proliferating natural killer cell

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3196301B1 (en) * 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of monogenic diseases
JP7103750B2 (ja) * 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
SG10201913782UA (en) * 2014-07-21 2020-03-30 Novartis Ag Treatment of cancer using a cll-1 chimeric antigen receptor
AU2015330699B2 (en) * 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
BR112017024514A2 (pt) * 2015-05-16 2018-07-24 Genzyme Corporation edição genética de mutações intrônicas profundas
CN108026526B (zh) * 2015-06-09 2023-05-12 爱迪塔斯医药公司 用于改善移植的crispr/cas相关方法和组合物
CN109715801B (zh) * 2015-12-01 2022-11-01 克里斯普治疗股份公司 用于治疗α1抗胰蛋白酶缺乏的材料和方法
AU2017347854B2 (en) 2016-10-27 2022-12-08 Intima Bioscience, Inc. Viral methods of T cell therapy
US11655275B2 (en) * 2017-05-03 2023-05-23 Sangamo Therapeutics, Inc. Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
EP3645038B1 (en) * 2017-06-30 2026-02-18 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene

Also Published As

Publication number Publication date
JP2025160172A (ja) 2025-10-22
IL286701A (en) 2021-10-31
KR20220017887A (ko) 2022-02-14
AU2020248097A1 (en) 2021-11-18
MX2021011782A (es) 2021-12-10
EP3946432A1 (en) 2022-02-09
CO2021014353A2 (es) 2022-01-17
US20220177917A1 (en) 2022-06-09
CN113891727A (zh) 2022-01-04
PH12021552383A1 (en) 2022-06-13
JP2022527709A (ja) 2022-06-03
BR112021019180A2 (pt) 2022-03-03
WO2020198675A1 (en) 2020-10-01
EP3946432A4 (en) 2023-04-05
JP7702878B2 (ja) 2025-07-04
SG11202110613SA (en) 2021-10-28

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