CO2021014353A2 - Generación de células nk primarias del receptor de antígeno quimérico (car) para la inmunoterapia al cáncer usando una combinación de virus cas9/rnp y aav - Google Patents

Generación de células nk primarias del receptor de antígeno quimérico (car) para la inmunoterapia al cáncer usando una combinación de virus cas9/rnp y aav

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Publication number
CO2021014353A2
CO2021014353A2 CONC2021/0014353A CO2021014353A CO2021014353A2 CO 2021014353 A2 CO2021014353 A2 CO 2021014353A2 CO 2021014353 A CO2021014353 A CO 2021014353A CO 2021014353 A2 CO2021014353 A2 CO 2021014353A2
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Colombia
Prior art keywords
cas9
cells
rnp
car
primary
Prior art date
Application number
CONC2021/0014353A
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English (en)
Inventor
Dean Anthony Lee
Kathrin Christine Meyer
Meisam Naeimi Kararoudi
Shibi Likhite
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Res Inst Nationwide Childrens Hospital
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Filing date
Publication date
Application filed by Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of CO2021014353A2 publication Critical patent/CO2021014353A2/es

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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
    • C12N15/63—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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/461—Cellular immunotherapy characterised by the cell type used
    • A61K39/4613—Natural-killer cells [NK or NK-T]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/463—Cellular immunotherapy characterised by recombinant expression
    • A61K39/4631—Chimeric Antigen Receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/4644—Cancer antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/4644—Cancer antigens
    • A61K39/464499—Undefined tumor antigens, e.g. tumor lysate or antigens targeted by cells isolated from tumor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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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    • C12N15/09—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
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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
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • 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 [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
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  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Microbiology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Biochemistry (AREA)
  • Mycology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)

Abstract

Se describen métodos para manipular células mediante ingeniería genética que utilizan el suministro viral adenoasociado (AAV) de un sistema CRISPR/CAS9 y ribonucleoproteínas para la integración. En algunos aspectos, en el presente documento se describen plásmidos de AAV para realizar dichos métodos.
CONC2021/0014353A 2019-03-27 2021-10-26 Generación de células nk primarias del receptor de antígeno quimérico (car) para la inmunoterapia al cáncer usando una combinación de virus cas9/rnp y aav CO2021014353A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962825007P 2019-03-27 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
CO2021014353A2 true CO2021014353A2 (es) 2022-01-17

Family

ID=72611833

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0014353A CO2021014353A2 (es) 2019-03-27 2021-10-26 Generación de células nk primarias del receptor de antígeno quimérico (car) para la inmunoterapia al cáncer usando una combinación de virus cas9/rnp y aav

Country Status (13)

Country Link
US (1) US20220177917A1 (es)
EP (1) EP3946432A4 (es)
JP (1) JP2022527709A (es)
KR (1) KR20220017887A (es)
CN (1) CN113891727A (es)
AU (1) AU2020248097A1 (es)
BR (1) BR112021019180A2 (es)
CA (1) CA3135198A1 (es)
CO (1) CO2021014353A2 (es)
IL (1) IL286701A (es)
MX (1) MX2021011782A (es)
SG (1) SG11202110613SA (es)
WO (1) WO2020198675A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL302335A (en) * 2020-10-26 2023-06-01 Res Inst Nationwide Childrens Hospital NK cells with chimeric antigen receptor (CAR) and uses thereof
WO2022236174A1 (en) * 2021-05-07 2022-11-10 Washington University In situ car-t therapies, vectors and methods therefor

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
WO2015089462A1 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
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
CN107849547B (zh) * 2015-05-16 2022-04-19 建新公司 深内含子突变的基因编辑
JP7396783B2 (ja) * 2015-06-09 2023-12-12 エディタス・メディシン、インコーポレイテッド 移植を改善するためのｃｒｉｓｐｒ／ｃａｓ関連方法および組成物
EP3384024B1 (en) * 2015-12-01 2022-02-02 CRISPR Therapeutics AG Materials and methods for treatment of alpha-1 antitrypsin deficiency
WO2018081476A2 (en) * 2016-10-27 2018-05-03 Intima Bioscience, Inc. Viral methods of t cell therapy
WO2018204469A2 (en) * 2017-05-03 2018-11-08 Sangamo Therapeutics, Inc. Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (cftr) gene
JP2020529834A (ja) * 2017-06-30 2020-10-15 プレシジョン バイオサイエンシズ，インク． Ｔ細胞受容体アルファ遺伝子の改変されたイントロンを含む遺伝子改変ｔ細胞

Also Published As

Publication number Publication date
WO2020198675A1 (en) 2020-10-01
KR20220017887A (ko) 2022-02-14
JP2022527709A (ja) 2022-06-03
CN113891727A (zh) 2022-01-04
AU2020248097A1 (en) 2021-11-18
IL286701A (en) 2021-10-31
BR112021019180A2 (pt) 2022-03-03
EP3946432A1 (en) 2022-02-09
US20220177917A1 (en) 2022-06-09
SG11202110613SA (en) 2021-10-28
CA3135198A1 (en) 2020-10-01
MX2021011782A (es) 2021-12-10
EP3946432A4 (en) 2023-04-05

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