CA3091491A1 - Expression of human foxp3 in gene edited t cells - Google Patents

Expression of human foxp3 in gene edited t cells Download PDF

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Publication number
CA3091491A1
CA3091491A1 CA3091491A CA3091491A CA3091491A1 CA 3091491 A1 CA3091491 A1 CA 3091491A1 CA 3091491 A CA3091491 A CA 3091491A CA 3091491 A CA3091491 A CA 3091491A CA 3091491 A1 CA3091491 A1 CA 3091491A1
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Canada
Prior art keywords
sequence
foxp3
nucleic acid
cell
seq
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Pending
Application number
CA3091491A
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English (en)
French (fr)
Inventor
Andrew M. Scharenberg
David J. Rawlings
Karen SOMMER
Yuchi Chiang HONAKER
Iram F. KHAN
Troy TORGERSON
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Seattle Childrens Hospital
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Seattle Childrens Hospital
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Publication of CA3091491A1 publication Critical patent/CA3091491A1/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
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    • A61P37/00—Drugs for immunological or allergic disorders
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    • 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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    • C12N15/09—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
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    • 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
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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
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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
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    • 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
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    • 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
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    • 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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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CA3091491A 2018-04-27 2019-04-25 Expression of human foxp3 in gene edited t cells Pending CA3091491A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862663561P 2018-04-27 2018-04-27
US62/663,561 2018-04-27
US201862773414P 2018-11-30 2018-11-30
US62/773,414 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
CA3091491A1 true CA3091491A1 (en) 2019-10-31

Family

ID=68295504

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3091491A Pending CA3091491A1 (en) 2018-04-27 2019-04-25 Expression of human foxp3 in gene edited t cells

Country Status (10)

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US (1) US20210253652A1 (https=)
EP (1) EP3784689A4 (https=)
JP (2) JP7575949B2 (https=)
KR (1) KR102912481B1 (https=)
CN (1) CN112041334A (https=)
AU (2) AU2019257708B2 (https=)
CA (1) CA3091491A1 (https=)
IL (1) IL277036A (https=)
SG (1) SG11202007877SA (https=)
WO (1) WO2019210078A1 (https=)

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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
US11713459B2 (en) 2018-04-27 2023-08-01 Seattle Children's Hospital Expression of FOXP3 in edited CD34+ cells
EP3921427A1 (en) * 2019-02-05 2021-12-15 Institut National de la Santé et de la Recherche Médicale (INSERM) Recombinant vectors suitable for the treatment of ipex syndrome
EP3973067A1 (en) * 2019-05-21 2022-03-30 Sangamo Therapeutics, Inc. Controlled transgene expression in regulatory t cells
US20220380727A1 (en) * 2019-11-08 2022-12-01 Sangamo Therapeutics, Inc. Generation of engineered regulatory t cells
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
EP4240376B1 (en) * 2020-11-09 2026-03-25 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
EP4634383A1 (en) * 2022-12-16 2025-10-22 Life Edit Therapeutics, Inc. Guide rnas that target foxp3 gene and methods of use
AU2024308500A1 (en) * 2023-06-24 2025-12-18 Sonoma Biotherapeutics, Inc. Compositions and methods for engineering cells
WO2025054503A1 (en) * 2023-09-08 2025-03-13 Sonoma Biotherapeutics, Inc. Cells ectopically expressing foxp3
JP7818147B1 (ja) * 2025-01-23 2026-02-20 国立研究開発法人国立がん研究センター エフェクターｔ細胞、およびそれを含む医薬組成物

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WO2008101272A1 (en) * 2007-02-21 2008-08-28 Women's And Children's Health Research Institute Inc 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
EP3294342A4 (en) * 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
WO2018035141A1 (en) * 2016-08-16 2018-02-22 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
CA3041831A1 (en) * 2016-10-27 2018-05-03 Intima Bioscience, Inc. Viral methods of making genetically modified cells
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
EP3672617A4 (en) * 2017-08-22 2021-06-23 The Regents of the University of California LENTIVIRAL VECTORS EXPRESSING FOXP3 IN HEMATOPOIETIC STEM CELLS TO TREAT IMMUNITY DEFICIENCIES AND AUTOIMMUNE DISEASES
US11713459B2 (en) * 2018-04-27 2023-08-01 Seattle Children's Hospital Expression of FOXP3 in edited CD34+ cells

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Publication number Publication date
JP7575949B2 (ja) 2024-10-31
IL277036A (en) 2020-10-29
AU2019257708B2 (en) 2024-08-22
US20210253652A1 (en) 2021-08-19
KR102912481B1 (ko) 2026-01-15
EP3784689A1 (en) 2021-03-03
JP2025013879A (ja) 2025-01-28
EP3784689A4 (en) 2022-01-19
KR20210005146A (ko) 2021-01-13
CN112041334A (zh) 2020-12-04
JP7844581B2 (ja) 2026-04-13
AU2019257708A1 (en) 2020-09-03
SG11202007877SA (en) 2020-09-29
AU2024266825A1 (en) 2024-12-12
WO2019210078A1 (en) 2019-10-31
JP2021521849A (ja) 2021-08-30

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