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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cell
cells
transgene
plasmid
aav
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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 Children's Hospital
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Nationwide Children's Hospital
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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
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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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    • C12N15/11—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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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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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
    • C12N2501/23—Interleukins [IL]
    • C12N2501/2321—Interleukin-21 (IL-21)
    • C—CHEMISTRY; METALLURGY
    • 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)
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  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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

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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) US12655449B2 (enExample)
EP (1) EP3946432A4 (enExample)
JP (2) JP7702878B2 (enExample)
KR (1) KR20220017887A (enExample)
CN (1) CN113891727A (enExample)
AU (1) AU2020248097B2 (enExample)
BR (1) BR112021019180A2 (enExample)
CA (1) CA3135198A1 (enExample)
CO (1) CO2021014353A2 (enExample)
IL (1) IL286701A (enExample)
MX (1) MX2021011782A (enExample)
PH (1) PH12021552383A1 (enExample)
SG (1) SG11202110613SA (enExample)
WO (1) WO2020198675A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019165121A1 (en) 2018-02-21 2019-08-29 Board Of Regents, The University Of Texas System Methods for activation and expansion of natural killer cells and uses therof
AU2021372458A1 (en) * 2020-10-26 2023-06-15 Research Institute At Nationwide Children's Hospital Chimeric antigen receptor (car) nk cells and uses thereof
WO2022236174A1 (en) * 2021-05-07 2022-11-10 Washington University In situ car-t therapies, vectors and methods therefor
CN117716024A (zh) * 2021-06-01 2024-03-15 瓦克斯细胞生物 包含饲养细胞的自然杀伤细胞增殖用组合物

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE051612T2 (hu) * 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
CN111206032B (zh) 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
BR112017000939A2 (pt) 2014-07-21 2017-11-14 Novartis Ag tratamento de câncer usando um receptor antigênico quimérico de cll-1
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
PL3298134T3 (pl) 2015-05-16 2023-09-18 Genzyme Corporation Edycja genów mutacji głęboko intronowych
US11911415B2 (en) * 2015-06-09 2024-02-27 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for improving transplantation
WO2017093804A2 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
CN110267978A (zh) 2016-10-07 2019-09-20 得克萨斯大学体系董事会 用于治疗癌症的表达膜锚定的il-12的t细胞
EP3532075A4 (en) 2016-10-27 2020-07-08 Intima Bioscience, Inc. VIRAL METHODS OF MANUFACTURING GENETICALLY MODIFIED CELLS
JP2020518245A (ja) 2017-05-03 2020-06-25 サンガモ セラピューティクス， インコーポレイテッド 嚢胞性線維症膜貫通コンダクタンス制御因子（ｃｆｔｒ）遺伝子の改変のための方法および組成物
EP4775669A2 (en) * 2017-06-30 2026-07-15 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
SG11202007288VA (en) 2018-01-30 2020-08-28 Res Institute At Nationwide Children’S Hospital Transforming growth factor beta-resistant natural killer cells
CN108504685A (zh) * 2018-03-27 2018-09-07 宜明细胞生物科技有限公司 一种利用CRISPR/Cas9系统同源重组修复IL-2RG缺陷基因的方法
AU2019271366B2 (en) 2018-05-16 2026-02-26 Research Institute At Nationwide Children's Hospital Generation of knock-out primary and expanded human NK cells using Cas9 ribonucleoproteins

Also Published As

Publication number Publication date
CO2021014353A2 (es) 2022-01-17
AU2020248097A1 (en) 2021-11-18
PH12021552383A1 (en) 2022-06-13
JP2022527709A (ja) 2022-06-03
US12655449B2 (en) 2026-06-16
BR112021019180A2 (pt) 2022-03-03
MX2021011782A (es) 2021-12-10
EP3946432A4 (en) 2023-04-05
US20220177917A1 (en) 2022-06-09
SG11202110613SA (en) 2021-10-28
AU2020248097B2 (en) 2026-05-21
IL286701A (en) 2021-10-31
EP3946432A1 (en) 2022-02-09
JP7702878B2 (ja) 2025-07-04
WO2020198675A1 (en) 2020-10-01
JP2025160172A (ja) 2025-10-22
KR20220017887A (ko) 2022-02-14
CN113891727A (zh) 2022-01-04

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