JP2020505044A - Ｂ細胞操作 - Google Patents

Ｂ細胞操作 Download PDF

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Publication number
JP2020505044A
JP2020505044A JP2019540095A JP2019540095A JP2020505044A JP 2020505044 A JP2020505044 A JP 2020505044A JP 2019540095 A JP2019540095 A JP 2019540095A JP 2019540095 A JP2019540095 A JP 2019540095A JP 2020505044 A JP2020505044 A JP 2020505044A
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JP
Japan
Prior art keywords
cells
cell
protein
transgene
genetically modified
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Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
JP2019540095A
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English (en)
Japanese (ja)
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JP2020505044A5 (enExample
Inventor
レーニア アモラ，
レーニア アモラ，
マイケル シー． ホームズ，
マイケル シー． ホームズ，
ブリジット イー． レイリー，
ブリジット イー． レイリー，
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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Publication date
Application filed by Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of JP2020505044A publication Critical patent/JP2020505044A/ja
Publication of JP2020505044A5 publication Critical patent/JP2020505044A5/ja
Priority to JP2022173206A priority Critical patent/JP2022191524A/ja
Withdrawn legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C12N5/12—Fused cells, e.g. hybridomas
    • C12N5/16—Animal cells
    • C12N5/163—Animal cells one of the fusion partners being a B or a T lymphocyte
    • 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
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • A61K40/13—B-cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/22—Immunosuppressive or immunotolerising
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/24—Antigen-presenting cells [APC]
    • 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
    • A61K40/416—Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N5/0635—B lymphocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Cell Biology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (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)
JP2019540095A 2017-01-26 2018-01-25 Ｂ細胞操作 Withdrawn JP2020505044A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022173206A JP2022191524A (ja) 2017-01-26 2022-10-28 Ｂ細胞操作

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762450917P 2017-01-26 2017-01-26
US62/450,917 2017-01-26
PCT/US2018/015180 WO2018140573A1 (en) 2017-01-26 2018-01-25 B-cell engineering

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022173206A Division JP2022191524A (ja) 2017-01-26 2022-10-28 Ｂ細胞操作

Publications (2)

Publication Number Publication Date
JP2020505044A true JP2020505044A (ja) 2020-02-20
JP2020505044A5 JP2020505044A5 (enExample) 2020-12-17

Family

ID=62979713

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019540095A Withdrawn JP2020505044A (ja) 2017-01-26 2018-01-25 Ｂ細胞操作
JP2022173206A Pending JP2022191524A (ja) 2017-01-26 2022-10-28 Ｂ細胞操作

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022173206A Pending JP2022191524A (ja) 2017-01-26 2022-10-28 Ｂ細胞操作

Country Status (11)

Country Link
US (1) US20190352614A1 (enExample)
EP (1) EP3573464A4 (enExample)
JP (2) JP2020505044A (enExample)
KR (1) KR20190111063A (enExample)
CN (1) CN110536963A (enExample)
AU (1) AU2018212652B2 (enExample)
BR (1) BR112019015355A2 (enExample)
CA (1) CA3051113A1 (enExample)
IL (1) IL268110B2 (enExample)
MX (1) MX2019008844A (enExample)
WO (1) WO2018140573A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
CA2935960C (en) 2014-01-08 2023-01-10 Bart Lipkens Acoustophoresis device with dual acoustophoretic chamber
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
EP3573464A4 (en) * 2017-01-26 2020-12-30 Sangamo Therapeutics, Inc. B CELL ENGINEERING
AU2018235957B2 (en) * 2017-03-16 2024-02-01 Seattle Children's Hospital (dba Seattle Children's Research Institute) Engraftable cell-based immunotherapy for long-term delivery of therapeutic proteins
KR20220066413A (ko) 2017-12-14 2022-05-24 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
BR112021007301A2 (pt) 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
WO2021085504A1 (ja) * 2019-11-01 2021-05-06 京都府公立大学法人 Ｂ細胞抗体受容体、及びその利用
EP4127188A4 (en) 2020-03-31 2024-08-21 Walking Fish Therapeutics MODIFIED B CELLS AND METHODS OF USE THEREOF
WO2023192936A2 (en) * 2022-03-30 2023-10-05 Fred Hutchinson Cancer Center Systems and methods to produce b cells that express selected antibodies and gene products
WO2024151683A2 (en) * 2023-01-09 2024-07-18 Walking Fish Therapeutics, Inc. HUMAN B CELL ENGINEERED TO EXPRESS IgA AND IgM ANTIBODIES FOR THERAPEUTIC USE
WO2024196669A2 (en) * 2023-03-17 2024-09-26 Walking Fish Therapeutics, Inc. Methods for in vivo editing of b cells
WO2024220447A2 (en) * 2023-04-17 2024-10-24 Be Biopharma, Inc. Engineered cell preparations for treatment of niemann pick b disease

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016161446A1 (en) * 2015-04-03 2016-10-06 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
WO2016210223A1 (en) * 2015-06-24 2016-12-29 Janssen Biotech, Inc. Immune modulation and treatment of solid tumors with antibodies that specifically bind cd38
WO2017011519A1 (en) * 2015-07-13 2017-01-19 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003901511A0 (en) * 2003-03-28 2003-04-17 Bionomics Limited Nucleic acid molecules associated with angiogenesis II
DK1804839T3 (da) * 2004-09-22 2012-04-10 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
US9834787B2 (en) * 2009-04-09 2017-12-05 Sangamo Therapeutics, Inc. Targeted integration into stem cells
AU2014265331B2 (en) * 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
US9757420B2 (en) * 2014-07-25 2017-09-12 Sangamo Therapeutics, Inc. Gene editing for HIV gene therapy
CN106795488B (zh) * 2014-09-16 2021-03-30 桑格摩治疗股份有限公司 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物
EP4177336A1 (en) * 2014-12-19 2023-05-10 Immusoft Corporation B cells for in vivo delivery of therapeutic agents
EP3402533B1 (en) * 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
EP3573464A4 (en) * 2017-01-26 2020-12-30 Sangamo Therapeutics, Inc. B CELL ENGINEERING
CN111533799A (zh) * 2017-04-10 2020-08-14 伊玛提克斯生物技术有限公司 用于白血病和其他癌症免疫治疗的肽和肽组合物
WO2018232296A1 (en) * 2017-06-16 2018-12-20 Sangamo Therapeutics, Inc. Targeted disruption of t cell and/or hla receptors
MX2020001594A (es) * 2017-08-08 2020-12-09 Sangamo Therapeutics Inc Direccionamiento celular mediado por receptor de antigeno quimerico.
CN111565730B (zh) * 2017-11-09 2024-09-17 桑格摩生物治疗股份有限公司 细胞因子诱导型含sh2蛋白（cish）基因的遗传修饰
US20230158070A1 (en) * 2018-01-30 2023-05-25 Cellectis Combination comprising allogeneic immune cells deficient for an antigen present on both t-cells and pathological cells and therapeutic antibody against said antigen
AU2019327913A1 (en) * 2018-08-29 2021-03-18 Nanjing Legend Biotech Co., Ltd. Anti-mesothelin chimeric antigen receptor (CAR) constructs and uses thereof
CN113748126A (zh) * 2018-11-30 2021-12-03 因提玛生物科学公司 鉴定免疫调节基因的方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016161446A1 (en) * 2015-04-03 2016-10-06 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
WO2016210223A1 (en) * 2015-06-24 2016-12-29 Janssen Biotech, Inc. Immune modulation and treatment of solid tumors with antibodies that specifically bind cd38
WO2017011519A1 (en) * 2015-07-13 2017-01-19 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
METHODS MOL. BIOL., vol. 271, JPN6021034270, 2004, pages 67 - 76, ISSN: 0004812158 *

Also Published As

Publication number Publication date
US20190352614A1 (en) 2019-11-21
IL268110B2 (en) 2024-01-01
IL268110B1 (en) 2023-09-01
KR20190111063A (ko) 2019-10-01
EP3573464A4 (en) 2020-12-30
JP2022191524A (ja) 2022-12-27
WO2018140573A1 (en) 2018-08-02
AU2018212652B2 (en) 2024-03-28
CA3051113A1 (en) 2018-08-02
EP3573464A1 (en) 2019-12-04
RU2019126606A3 (enExample) 2021-06-11
IL268110A (en) 2019-09-26
BR112019015355A2 (pt) 2020-05-19
AU2018212652A1 (en) 2019-08-01
CN110536963A (zh) 2019-12-03
MX2019008844A (es) 2019-09-10
RU2019126606A (ru) 2021-02-26

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