CA3156509A1 - Generation of cd38 knock-out primary and expanded human nk cells - Google Patents

Generation of cd38 knock-out primary and expanded human nk cells Download PDF

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Publication number
CA3156509A1
CA3156509A1 CA3156509A CA3156509A CA3156509A1 CA 3156509 A1 CA3156509 A1 CA 3156509A1 CA 3156509 A CA3156509 A CA 3156509A CA 3156509 A CA3156509 A CA 3156509A CA 3156509 A1 CA3156509 A1 CA 3156509A1
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Prior art keywords
cells
cell
cancer
subject
sequence
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Pending
Application number
CA3156509A
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English (en)
French (fr)
Inventor
Dean Anthony LEE
Meisam Naeimi KARAROUDI
Gabriel Ghiaur
Yuya Nagai
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Johns Hopkins University
Nationwide Childrens Hospital Inc
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Johns Hopkins University
Nationwide Childrens Hospital Inc
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Publication of CA3156509A1 publication Critical patent/CA3156509A1/en
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    • 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
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/39558—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against tumor tissues, cells, antigens
    • 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
    • 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
    • A61K40/40—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
    • A61K40/4222—CD38 not IgG
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2896—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against molecules with a "CD"-designation, not provided for elsewhere
    • 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
    • C12N5/0646—Natural killers cells [NK], NKT cells
    • 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
    • A61K2239/46—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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/73—Inducing cell death, e.g. apoptosis, necrosis or inhibition of cell proliferation
    • C07K2317/732—Antibody-dependent cellular cytotoxicity [ADCC]
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hematology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Oncology (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3156509A 2019-10-31 2020-11-02 Generation of cd38 knock-out primary and expanded human nk cells Pending CA3156509A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962928524P 2019-10-31 2019-10-31
US62/928,524 2019-10-31
PCT/US2020/058565 WO2021087466A1 (en) 2019-10-31 2020-11-02 Generation of cd38 knock-out primary and expanded human nk cells

Publications (1)

Publication Number Publication Date
CA3156509A1 true CA3156509A1 (en) 2021-05-06

Family

ID=75715400

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3156509A Pending CA3156509A1 (en) 2019-10-31 2020-11-02 Generation of cd38 knock-out primary and expanded human nk cells

Country Status (11)

Country Link
US (1) US20220401482A1 (enExample)
EP (1) EP4051297A4 (enExample)
JP (1) JP2022554284A (enExample)
KR (1) KR20220093337A (enExample)
CN (1) CN114867485A (enExample)
AU (1) AU2020375053A1 (enExample)
BR (1) BR112022008215A2 (enExample)
CA (1) CA3156509A1 (enExample)
IL (1) IL292584A (enExample)
MX (1) MX2022005051A (enExample)
WO (1) WO2021087466A1 (enExample)

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Publication number Priority date Publication date Assignee Title
TW202237827A (zh) 2020-12-03 2022-10-01 美商世紀治療股份有限公司 經基因工程改造之細胞及其用途
US11661459B2 (en) 2020-12-03 2023-05-30 Century Therapeutics, Inc. Artificial cell death polypeptide for chimeric antigen receptor and uses thereof
JP2024500847A (ja) 2020-12-18 2024-01-10 センチュリー セラピューティクス，インコーポレイテッド 適合可能な受容体特異性を有するキメラ抗原受容体システム
EP4347816A4 (en) * 2021-06-02 2026-04-29 Metagenomi Therapeutics Inc CRISPR CLASS II, TYPE V SYSTEMS
AU2022301302A1 (en) 2021-07-01 2024-01-25 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
US20250302874A1 (en) * 2021-11-03 2025-10-02 Intellia Therapeutics, Inc. Cd38 compositions and methods for immunotherapy
JP2025001053A (ja) * 2021-11-05 2025-01-08 学校法人東海大学 劇症型ｎｋ白血病に対する治療標的の同定
JP2025509343A (ja) 2022-03-07 2025-04-11 サノフィ－アベンティス・ユー・エス・エルエルシー 多発性骨髄腫の治療のための他の薬剤と組み合わせたイサツキシマブの使用
CN114921416B (zh) * 2022-05-12 2023-05-23 广东普罗凯融生物医药科技有限公司 一种nk细胞及其制备方法
CN120051297A (zh) 2022-06-30 2025-05-27 因达普塔治疗公司 工程化自然杀伤(nk)细胞与抗体疗法的组合及相关方法
WO2024124244A1 (en) * 2022-12-09 2024-06-13 Research Institute At Nationwide Children's Hospital Cd38 as integration site for enhanced function of gene-modified immune cells
WO2024124242A2 (en) * 2022-12-09 2024-06-13 Research Institute At Nationwide Children's Hospital Combination cd38ko/car ki nk cell immunotherapy for dual targeting with cd38 monoclonal antibodies
WO2024155711A1 (en) * 2023-01-17 2024-07-25 Research Institute At Nationwide Children's Hospital Methods of targeting glycogen synthase kinase 3 beta in nk cells
CN116679065B (zh) * 2023-07-31 2023-11-14 北京大学人民医院 检测试剂的应用、多发性骨髓瘤治疗预后预测方法及产品
WO2025213127A1 (en) 2024-04-05 2025-10-09 Indapta Therapeutics, Inc. Treatment of autoimmune diseases or conditions with natural killer cells
WO2025245399A1 (en) 2024-05-23 2025-11-27 Indapta Therapeutics, Inc. Method of treating cancer with natural killer cells
WO2026050706A1 (en) * 2024-08-30 2026-03-05 Aventis Inc. Nk cells having reduced cd38 expression and method for producing

Family Cites Families (13)

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US128A (en) 1837-02-16 Holdback for sleds
FR1601438A (enExample) 1968-10-17 1970-08-24
CA2926078C (en) * 2013-10-17 2021-11-16 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
US10106620B2 (en) * 2014-06-16 2018-10-23 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Blocking CD38 using anti-CD38 F(ab′)2 to protect NK cells
AU2015289644A1 (en) * 2014-07-15 2017-02-02 Juno Therapeutics, Inc. Engineered cells for adoptive cell therapy
US10876134B2 (en) * 2014-12-16 2020-12-29 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
EP3317399B1 (en) * 2015-06-30 2024-06-26 Cellectis Methods for improving functionality in nk cell by gene inactivation using specific endonuclease
MA42895A (fr) * 2015-07-15 2018-05-23 Juno Therapeutics Inc Cellules modifiées pour thérapie cellulaire adoptive
EP3405568A4 (en) * 2016-01-20 2019-12-04 Fate Therapeutics, Inc. COMPOUNDS AND METHODS FOR IMMUNOCELL MODULATION IN ADOPTIVE IMMUNOTHERAPIES
CA3020330A1 (en) * 2016-04-07 2017-10-12 Bluebird Bio, Inc. Chimeric antigen receptor t cell compositions
ES2875005T3 (es) * 2016-12-09 2021-11-08 Onk Therapeutics Ltd Células asesinas naturales manipuladas y usos de las mismas
KR20250048610A (ko) * 2017-12-22 2025-04-09 페이트 세러퓨틱스, 인코포레이티드 향상된 면역 효과기 세포 및 이의 용도
CA3100341A1 (en) * 2018-05-16 2019-11-21 Research Institute Of Nationwide Children's Hospital Generation of knock-out primary and expanded human nk cells using cas9 ribonucleoproteins

Also Published As

Publication number Publication date
KR20220093337A (ko) 2022-07-05
IL292584A (en) 2022-06-01
MX2022005051A (es) 2022-08-08
EP4051297A4 (en) 2023-10-18
AU2020375053A1 (en) 2022-05-26
EP4051297A1 (en) 2022-09-07
US20220401482A1 (en) 2022-12-22
JP2022554284A (ja) 2022-12-28
WO2021087466A1 (en) 2021-05-06
BR112022008215A2 (pt) 2022-07-12
CN114867485A (zh) 2022-08-05

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