SG11202007877SA - Expression of human foxp3 in gene edited t cells - Google Patents

Expression of human foxp3 in gene edited t cells

Info

Publication number
SG11202007877SA
SG11202007877SA SG11202007877SA SG11202007877SA SG11202007877SA SG 11202007877S A SG11202007877S A SG 11202007877SA SG 11202007877S A SG11202007877S A SG 11202007877SA SG 11202007877S A SG11202007877S A SG 11202007877SA SG 11202007877S A SG11202007877S A SG 11202007877SA
Authority
SG
Singapore
Prior art keywords
expression
cells
gene edited
human foxp3
foxp3
Prior art date
Application number
SG11202007877SA
Other languages
English (en)
Inventor
Andrew M Scharenberg
David J Rawlings
Karen Sommer
Yuchi Chiang Honaker
Iram F Khan
Troy Torgerson
Original Assignee
Seattle Childrens Hospital Dba Seattle Childrens Res Inst
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 Seattle Childrens Hospital Dba Seattle Childrens Res Inst filed Critical Seattle Childrens Hospital Dba Seattle Childrens Res Inst
Publication of SG11202007877SA publication Critical patent/SG11202007877SA/en

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    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] 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
    • A61K40/40—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
    • 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
    • A61K40/418—Antigens related to induction of tolerance to non-self
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/87—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
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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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the route of administration
    • 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
    • A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
    • 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
    • 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 [CRISPR]
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • 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
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Developmental Biology & Embryology (AREA)
  • Transplantation (AREA)
  • Mycology (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
SG11202007877SA 2018-04-27 2019-04-25 Expression of human foxp3 in gene edited t cells SG11202007877SA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862663561P 2018-04-27 2018-04-27
US201862773414P 2018-11-30 2018-11-30
PCT/US2019/029159 WO2019210078A1 (en) 2018-04-27 2019-04-25 Expression of human foxp3 in gene edited t cells

Publications (1)

Publication Number Publication Date
SG11202007877SA true SG11202007877SA (en) 2020-09-29

Family

ID=68295504

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202007877SA SG11202007877SA (en) 2018-04-27 2019-04-25 Expression of human foxp3 in gene edited t cells

Country Status (10)

Country Link
US (1) US20210253652A1 (enExample)
EP (1) EP3784689A4 (enExample)
JP (2) JP7575949B2 (enExample)
KR (1) KR20210005146A (enExample)
CN (1) CN112041334A (enExample)
AU (2) AU2019257708B2 (enExample)
CA (1) CA3091491A1 (enExample)
IL (1) IL277036A (enExample)
SG (1) SG11202007877SA (enExample)
WO (1) WO2019210078A1 (enExample)

Families Citing this family (13)

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CN110139675B (zh) * 2016-10-31 2023-09-29 西雅图儿童医院（Dba西雅图儿童研究所） 用具有工程化稳定的内源性foxp3基因表达的cd4t细胞治疗自身免疫疾病的方法
WO2019210042A1 (en) 2018-04-27 2019-10-31 Seattle Children's Hospital (dba Seattle Children's Research Institute) Expression of foxp3 in edited cd34+ cells
WO2020161122A1 (en) * 2019-02-05 2020-08-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Recombinant vectors suitable for the treatment of ipex syndrome
CA3141159A1 (en) 2019-05-21 2020-11-26 Sangamo Therapeutics, Inc. Controlled transgene expression in regulatory t cells
JP2023501388A (ja) * 2019-11-08 2023-01-18 サンガモ セラピューティクス， インコーポレイテッド 操作された制御性ｔ細胞の生成
EP4103591A4 (en) * 2020-02-13 2024-09-04 The Board of Trustees of the Leland Stanford Junior University Crispr-based foxp3 gene engineered t cells and hematopoietic stem cell precursors to treat ipex syndrome patients
EP4240376A1 (en) * 2020-11-09 2023-09-13 Quell Therapeutics Limited Method for cryopreserving engineered tregs
EP4355879A4 (en) * 2021-06-14 2025-07-23 Kamau Therapeutics Inc METHODS FOR GENETIC MODIFICATION OF HEMATOPOIETIC STEM AND PROGENITOR CELLS FOR ERYTHROCYTE-SPECIFIC EXPRESSION OF THERAPEUTIC PROTEINS
WO2023122099A2 (en) * 2021-12-21 2023-06-29 The Board Of Trustees Of The Leland Stanford Junior University Crispr-based gene editing to preserve splicing and expression of foxp3 isoforms 1 and 2
CN115305237B (zh) * 2022-07-08 2023-07-14 中南大学 一种优化的调节性t细胞的扩增及规模化生产的方法
WO2024127369A1 (en) * 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target foxp3 gene and methods of use
WO2025006409A2 (en) * 2023-06-24 2025-01-02 Sonoma Biotherapeutics, Inc. Compositions and methods for engineering cells
WO2025054503A1 (en) * 2023-09-08 2025-03-13 Sonoma Biotherapeutics, Inc. Cells ectopically expressing foxp3

Family Cites Families (11)

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AU2007347364A1 (en) * 2007-02-21 2008-08-28 Adelaide Research & Innovation Pty Ltd Method for obtaining TREG-cells
ES2883131T3 (es) * 2013-05-29 2021-12-07 Cellectis Métodos para la modificación de células T para inmunoterapia utilizando el sistema de nucleasa CAS guiado por ARN
WO2016123578A1 (en) * 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
AU2016261600B2 (en) * 2015-05-08 2021-09-23 President And Fellows Of Harvard College Universal donor stem cells and related methods
US20190309274A1 (en) * 2016-08-16 2019-10-10 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
WO2018073391A1 (en) * 2016-10-19 2018-04-26 Cellectis Targeted gene insertion for improved immune cells therapy
GB2607227B (en) * 2016-10-27 2023-05-10 Intima Bioscience Inc Viral methods of T cell therapy
CN110139675B (zh) 2016-10-31 2023-09-29 西雅图儿童医院（Dba西雅图儿童研究所） 用具有工程化稳定的内源性foxp3基因表达的cd4t细胞治疗自身免疫疾病的方法
CN109797171A (zh) * 2017-05-08 2019-05-24 北京东方略细胞技术有限公司 经修饰的t细胞、其制备方法及用途
JP7290288B2 (ja) * 2017-08-22 2023-06-13 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 免疫不全及び自己免疫疾患を治療するための、造血幹細胞でｆｏｘｐ３を発現するレンチウイルスベクター
WO2019210042A1 (en) * 2018-04-27 2019-10-31 Seattle Children's Hospital (dba Seattle Children's Research Institute) Expression of foxp3 in edited cd34+ cells

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Publication number Publication date
EP3784689A1 (en) 2021-03-03
AU2024266825A1 (en) 2024-12-12
AU2019257708B2 (en) 2024-08-22
EP3784689A4 (en) 2022-01-19
WO2019210078A1 (en) 2019-10-31
CN112041334A (zh) 2020-12-04
JP2025013879A (ja) 2025-01-28
US20210253652A1 (en) 2021-08-19
CA3091491A1 (en) 2019-10-31
IL277036A (en) 2020-10-29
JP2021521849A (ja) 2021-08-30
JP7575949B2 (ja) 2024-10-31
AU2019257708A1 (en) 2020-09-03
KR20210005146A (ko) 2021-01-13

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