EP3735460A4 - Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof - Google Patents

Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof Download PDF

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Publication number
EP3735460A4
EP3735460A4 EP18856963.6A EP18856963A EP3735460A4 EP 3735460 A4 EP3735460 A4 EP 3735460A4 EP 18856963 A EP18856963 A EP 18856963A EP 3735460 A4 EP3735460 A4 EP 3735460A4
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EP
European Patent Office
Prior art keywords
regulating
compositions
activity
methods
genetically modifying
Prior art date
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.)
Pending
Application number
EP18856963.6A
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German (de)
French (fr)
Other versions
EP3735460A1 (en
Inventor
Gregory Ian Frost
James Joseph ONUFFER
Farzad Haerizadeh
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.)
Exuma Biotech Corp
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Exuma Biotech Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Priority claimed from PCT/US2018/020818 external-priority patent/WO2018161064A1/en
Application filed by Exuma Biotech Corp filed Critical Exuma Biotech Corp
Publication of EP3735460A1 publication Critical patent/EP3735460A1/en
Publication of EP3735460A4 publication Critical patent/EP3735460A4/en
Pending legal-status Critical Current

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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • A61K39/4611—T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
    • 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
    • A61K39/464402—Receptors, cell surface antigens or cell surface determinants
    • 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
    • A61K39/464402—Receptors, cell surface antigens or cell surface determinants
    • A61K39/464411—Immunoglobulin superfamily
    • A61K39/464412—CD19 or B4
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/70517—CD8
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/70532—B7 molecules, e.g. CD80, CD86
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
    • C07K14/7155—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons for interleukins [IL]
    • 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
    • C12N5/0636—T lymphocytes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/90—Isomerases (5.)
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/60—Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16045—Special targeting system for viral vectors
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16051—Methods of production or purification of viral material
    • C12N2740/16052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
    • C12N2830/205—Vector systems having a special element relevant for transcription transcription of more than one cistron bidirectional
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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EP18856963.6A 2017-09-18 2018-09-17 Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof Pending EP3735460A4 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201762560176P 2017-09-18 2017-09-18
US201762564253P 2017-09-27 2017-09-27
US201762564991P 2017-09-28 2017-09-28
PCT/US2018/020818 WO2018161064A1 (en) 2017-03-03 2018-03-03 Methods and compositions for transducing and expanding lymphocytes and regulating the activity thereof
US201862728056P 2018-09-06 2018-09-06
PCT/US2018/051392 WO2019055946A1 (en) 2017-09-18 2018-09-17 Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof

Publications (2)

Publication Number Publication Date
EP3735460A1 EP3735460A1 (en) 2020-11-11
EP3735460A4 true EP3735460A4 (en) 2021-08-11

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EP18856963.6A Pending EP3735460A4 (en) 2017-09-18 2018-09-17 Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof

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EP (1) EP3735460A4 (en)
CN (2) CN111479921B (en)
TW (1) TW201920250A (en)
WO (1) WO2019055946A1 (en)

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US11111505B2 (en) 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
CA3111084A1 (en) * 2018-09-02 2020-03-05 Exuma Biotech, Corp Methods and compositions for genetically modifying lymphocytes in blood or in enriched pbmcs
CN113508128A (en) 2019-03-01 2021-10-15 艾洛基治疗公司 Constitutively active chimeric cytokine receptors
WO2020180664A1 (en) 2019-03-01 2020-09-10 Allogene Therapeutics, Inc. Chimeric cytokine receptors bearing a pd-1 ectodomain
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AU2020336230A1 (en) * 2019-09-01 2022-03-31 Exuma Biotech Corp. Methods and compositions for the modification and delivery of lymphocytes
JP2022546592A (en) 2019-09-03 2022-11-04 マイエロイド・セラピューティクス，インコーポレーテッド Methods and compositions for genomic integration
US20240122978A1 (en) * 2019-10-16 2024-04-18 Umoja Biopharma, Inc. Retroviral vector for univeral receptor therapy
GB201918586D0 (en) * 2019-12-17 2020-01-29 Patterson James Engineered platelets for targeted delivery of a therapeutic agent
CA3167065A1 (en) 2020-02-24 2021-09-02 Regina Junhui LIN Bcma car-t cells with enhanced activities
EP4114400A4 (en) * 2020-03-05 2023-10-18 Exuma Biotech Corp. Methods and compositions for the delivery of modified lymphocyte aggregates
CN113913392A (en) * 2020-07-10 2022-01-11 中国人民解放军军事科学院军事医学研究院 Method for constructing HCMV (human chorionic gonadotropin) coding miRNA (miRNA) deletion strain based on Crispr-cas9 technology and application of method
CN111849896B (en) * 2020-07-31 2022-07-01 广东昭泰体内生物医药科技有限公司 CAR-NK cell culture medium and application thereof
CN113201063B (en) * 2020-07-31 2022-04-19 北京市神经外科研究所 Truncation of IL7R alpha and application thereof in preparing medicament for treating tumor
TW202227636A (en) * 2020-09-08 2022-07-16 美商賽特免疫治療公司 Methods and compositions of infecting, activating, and expanding immune cells
EP4240367A4 (en) 2020-11-04 2024-10-16 Myeloid Therapeutics Inc Engineered chimeric fusion protein compositions and methods of use thereof
AU2022212952A1 (en) * 2021-01-27 2023-08-10 Umoja Biopharma, Inc. Lentivirus for generating cells expressing anti-cd19 chimeric antigen receptor
WO2022187289A1 (en) 2021-03-01 2022-09-09 Exuma Biotech Corp. Methods and compositions for the delivery of retroviral particles
CN112662631B (en) * 2021-03-16 2021-06-29 合源生物科技(天津)有限公司 CAR-T cell perfusion culture method
KR20240013094A (en) 2021-03-17 2024-01-30 마이얼로이드 테라퓨틱스, 인크. Engineered chimeric fusion protein compositions and methods of using the same
CN113249331B (en) * 2021-06-07 2021-10-22 北京翊博普惠生物科技发展有限公司 DC cell and CTL cell loaded with Tax antigen as well as preparation methods and applications of DC cell and CTL cell
WO2023081813A1 (en) * 2021-11-05 2023-05-11 St. Jude Children's Research Hospital, Inc. Zip cytokine receptors
WO2023088246A1 (en) * 2021-11-17 2023-05-25 上海君赛生物科技有限公司 Membrane surface protein containing gpi anchor region
EP4453022A1 (en) * 2021-12-22 2024-10-30 Quell Therapeutics Limited Constitutive cytokine receptors
WO2023168305A1 (en) 2022-03-01 2023-09-07 Exuma Biotech Corp. Viral particles with membrane-bound hyaluronidase
CN114875069B (en) * 2022-04-22 2023-09-15 四川大学 Recombinant vector of genetically modified IL2 cytokine, host cell and application thereof

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MAURICE M ET AL: "Efficient gene transfer into human primary blood lymphocytes by surface-engineered lentiviral vectors that display a T cell-activating polypeptide", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, US, vol. 99, no. 7, 1 April 2002 (2002-04-01), pages 2342 - 2350, XP002346143, ISSN: 0006-4971, DOI: 10.1182/BLOOD.V99.7.2342 *
See also references of WO2019055946A1 *

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CN111479921B (en) 2024-08-02
EP3735460A1 (en) 2020-11-11

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