BR112019007715A2 - inserção vetorizada de genes para terapia aprimorada com células imunes - Google Patents

inserção vetorizada de genes para terapia aprimorada com células imunes

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Publication number
BR112019007715A2
BR112019007715A2 BR112019007715A BR112019007715A BR112019007715A2 BR 112019007715 A2 BR112019007715 A2 BR 112019007715A2 BR 112019007715 A BR112019007715 A BR 112019007715A BR 112019007715 A BR112019007715 A BR 112019007715A BR 112019007715 A2 BR112019007715 A2 BR 112019007715A2
Authority
BR
Brazil
Prior art keywords
immune cell
vectorized
cell therapy
enhanced immune
gene insertion
Prior art date
Application number
BR112019007715A
Other languages
English (en)
Portuguese (pt)
Inventor
Juillerat Alexandre
Busser Brian
Valton Julien
Poirot Laurent
Duchateau Philippe
Original Assignee
Cellectis Sa
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
Application filed by Cellectis Sa filed Critical Cellectis Sa
Priority claimed from PCT/EP2017/076798 external-priority patent/WO2018073391A1/en
Publication of BR112019007715A2 publication Critical patent/BR112019007715A2/pt

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14Blood; Artificial blood
    • A61K35/17Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • A61K40/11T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31Chimeric antigen receptors [CAR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/36Immune checkpoint inhibitors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/4203Receptors for growth factors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/421Immunoglobulin superfamily
    • A61K40/4212CD22, BL-CAM, siglec-2 or sialic acid binding Ig-related lectin 2
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
    • C07K14/7051T-cell receptor (TcR)-CD3 complex
    • CCHEMISTRY; METALLURGY
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
    • C12N5/0638Cytotoxic T lymphocytes [CTL] or lymphokine activated killer cells [LAK]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/46Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the cancer treated
    • A61K2239/48Blood cells, e.g. leukemia or lymphoma
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Cell Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
BR112019007715A 2016-10-19 2017-10-19 inserção vetorizada de genes para terapia aprimorada com células imunes BR112019007715A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662410187P 2016-10-19 2016-10-19
DKPA201670840 2016-10-27
PCT/EP2017/076798 WO2018073391A1 (en) 2016-10-19 2017-10-19 Targeted gene insertion for improved immune cells therapy

Publications (1)

Publication Number Publication Date
BR112019007715A2 true BR112019007715A2 (pt) 2019-07-09

Family

ID=60268351

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019007715A BR112019007715A2 (pt) 2016-10-19 2017-10-19 inserção vetorizada de genes para terapia aprimorada com células imunes

Country Status (10)

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US (2) US11873511B2 (enExample)
EP (1) EP3529351A1 (enExample)
JP (1) JP7274416B2 (enExample)
KR (1) KR102709329B1 (enExample)
CN (1) CN110023490B (enExample)
AU (1) AU2017347637B2 (enExample)
BR (1) BR112019007715A2 (enExample)
CA (1) CA3040048A1 (enExample)
IL (1) IL265953A (enExample)
MX (1) MX2019004215A (enExample)

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CA2993201A1 (en) 2015-07-21 2017-01-26 The Children's Medical Center Corporation Pd-l1 expressing hematopoietic stem cells and uses
WO2019060708A1 (en) 2017-09-22 2019-03-28 The Children's Medical Center Corporation TREATMENT OF TYPE 1 DIABETES AND AUTOIMMUNE DISEASES OR DISORDERS
CN110841058B (zh) * 2019-11-21 2022-09-02 北京启辰生生物科技有限公司 用于协同改善免疫应答的组合物及应用
CN110790842B (zh) * 2019-11-25 2021-03-30 贵州康钦承平生物科技有限公司 一种FasL-CAR融合蛋白和表达融合蛋白的T细胞及其制备方法和应用
US20240216508A1 (en) * 2020-06-26 2024-07-04 Juno Therapeutics Gmbh Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods
US11591381B2 (en) 2020-11-30 2023-02-28 Crispr Therapeutics Ag Gene-edited natural killer cells
US11661459B2 (en) 2020-12-03 2023-05-30 Century Therapeutics, Inc. Artificial cell death polypeptide for chimeric antigen receptor and uses thereof
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
WO2022133169A1 (en) 2020-12-18 2022-06-23 Century Therapeutics, Inc. Chimeric antigen receptor systems with adaptable receptor specificity
US11473060B2 (en) 2020-12-30 2022-10-18 Crispr Therapeutics Ag Compositions and methods for differentiating stem cells into NK cells
KR102491095B1 (ko) * 2021-01-22 2023-01-20 한국생명공학연구원 Cas 단백질 및 HIF의 TAD 도메인을 포함하는 단백질 융합체 및 이의 용도
KR102500873B1 (ko) * 2021-02-03 2023-02-17 한국과학기술연구원 자연살해세포 특이적 CRISPR/Cas 시스템을 위한 융합 단백질 및 이의 용도
US20230016422A1 (en) * 2021-06-23 2023-01-19 Crispr Therapeutics Ag Engineered cells with improved protection from natural killer cell killing
CN113604437A (zh) * 2021-08-13 2021-11-05 青岛华赛伯曼医学细胞生物有限公司 过表达ccr2的免疫细胞及其应用
WO2023088437A1 (zh) * 2021-11-19 2023-05-25 南开大学 重组武装溶瘤病毒组合物及其在til过继治疗中的用途
WO2024022509A1 (en) * 2022-07-29 2024-02-01 Nanjing Legend Biotech Co., Ltd. Methods for promoting persistence of cell therapy
CN115820697B (zh) * 2022-09-23 2024-07-30 中国海洋大学 一种免疫细胞及其制备方法和应用
CN115286697B (zh) * 2022-09-29 2022-12-13 烟台蓝纳成生物技术有限公司 一种双重靶向化合物及其制备方法和应用
CN115851909B (zh) * 2022-11-21 2023-09-08 郑州大学第一附属医院 检测fosb基因的试剂在制备诊断腹主动脉瘤产品的应用

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HUE064187T2 (hu) * 2012-05-25 2024-02-28 Cellectis Eljárás immunterápiára alkalmas allogén és immunszupresszív-rezisztens T-sejt módosítására
CN105378067A (zh) * 2013-05-13 2016-03-02 塞勒克提斯公司 工程化用于免疫治疗的高活性t细胞的方法
AU2014273490B2 (en) 2013-05-29 2019-05-09 Cellectis Methods for engineering T cells for immunotherapy by using RNA-guided Cas nuclease system
JP6976058B2 (ja) 2013-11-22 2021-12-01 セレクティスCellectis 免疫療法のための化学療法薬耐性t細胞を工学的に作製する方法
EP3693384B1 (en) 2014-03-11 2024-01-24 Cellectis Method for generating t-cells compatible for allogenic transplantation
EP3253866B1 (en) 2015-02-06 2024-12-04 Cellectis Primary hematopoietic cells genetically engineered by slow release of nucleic acids using nanoparticles
EP4269601A3 (en) 2015-03-27 2024-01-10 President and Fellows of Harvard College Modified t cells and methods of making and using the same
IL257105B (en) * 2015-07-31 2022-09-01 Univ Minnesota Modified cells and methods of therapy
PT4180519T (pt) * 2016-04-15 2025-09-04 Memorial Sloan Kettering Cancer Center Células t transgénicas e composições de células t que expressam um recetor antigénico quimérico e métodos relacionados

Also Published As

Publication number Publication date
AU2017347637A1 (en) 2019-05-02
CA3040048A1 (en) 2018-04-26
JP7274416B2 (ja) 2023-05-16
CN110023490B (zh) 2024-08-09
US11873511B2 (en) 2024-01-16
KR102709329B1 (ko) 2024-09-23
MX2019004215A (es) 2019-08-21
RU2019113911A (ru) 2020-11-20
US20240141293A1 (en) 2024-05-02
CN110023490A (zh) 2019-07-16
JP2019531743A (ja) 2019-11-07
AU2017347637B2 (en) 2024-02-15
IL265953A (en) 2019-05-30
US12391933B2 (en) 2025-08-19
EP3529351A1 (en) 2019-08-28
US20200224163A1 (en) 2020-07-16
RU2019113911A3 (enExample) 2021-06-28
KR20190065397A (ko) 2019-06-11

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