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 Children's Hospital
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Seattle Children's 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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    • C12N15/87—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
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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
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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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    • 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 (enExample)
EP (1) EP3784689A4 (enExample)
JP (3) JP7575949B2 (enExample)
KR (1) KR102912481B1 (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)

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CA3042179A1 (en) 2016-10-31 2018-05-03 Seattle Children's Hospital (dba Seattle Children's Research Institute) Method for treating autoimmune disease using cd4 t-cells with engineered stabilization of expression of endogenous foxp3 gene
SG11202007878UA (en) 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst 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
JP2022533713A (ja) * 2019-05-21 2022-07-25 サンガモ セラピューティクス， インコーポレイテッド 調節性ｔ細胞における制御された導入遺伝子発現
CA3160113A1 (en) * 2019-11-08 2021-05-14 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
IL302591A (en) * 2020-11-09 2023-07-01 Quell Therapeutics Ltd Method for cryopreservation of transgenic regulatory T cells
WO2022266139A2 (en) * 2021-06-14 2022-12-22 Graphite Bio, Inc. Methods to genetically engineer hematopoietic stem and progenitor cells for red cell 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
CN121532517A (zh) * 2023-06-24 2026-02-13 索诺玛生物治疗公司 用于对细胞进行工程改造的组合物和方法
CN122055438A (zh) * 2023-09-08 2026-05-15 索诺玛生物治疗公司 异位表达foxp3的细胞
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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US20110123502A1 (en) 2007-02-21 2011-05-26 Barry Simon C Method for obtaining treg-cells
JP6608807B2 (ja) * 2013-05-29 2019-11-20 セレクティス Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法
US20190388469A1 (en) 2015-01-30 2019-12-26 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
EP3500696A4 (en) 2016-08-16 2020-04-08 Bluebird Bio, Inc. VARIANTS OF IL-10 ALPHA RECEPTOR HOMING ENDONUCLEASE, COMPOSITIONS AND METHODS OF USE
WO2018073391A1 (en) * 2016-10-19 2018-04-26 Cellectis Targeted gene insertion for improved immune cells therapy
EP3532075A4 (en) * 2016-10-27 2020-07-08 Intima Bioscience, Inc. VIRAL METHODS OF MANUFACTURING GENETICALLY MODIFIED CELLS
CA3042179A1 (en) 2016-10-31 2018-05-03 Seattle Children's Hospital (dba Seattle Children's Research Institute) Method for treating autoimmune disease using cd4 t-cells with engineered stabilization of expression of endogenous foxp3 gene
CN109790518A (zh) * 2017-05-08 2019-05-21 中国科学院动物研究所 经修饰的t细胞、其制备方法及用途
WO2019040655A1 (en) * 2017-08-22 2019-02-28 The Regents Of The University Of California LENTIVIRAL VECTORS EXPRESSING FOXP3 IN HEMATOPOIETIC STEM CELLS TO TREAT IMMUNITY DEFICIENCIES AND AUTOIMMUNE DISEASES
SG11202007878UA (en) * 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Expression of foxp3 in edited cd34+ cells

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Publication number Publication date
US20210253652A1 (en) 2021-08-19
JP7575949B2 (ja) 2024-10-31
JP7844581B2 (ja) 2026-04-13
JP2025013879A (ja) 2025-01-28
AU2019257708B2 (en) 2024-08-22
SG11202007877SA (en) 2020-09-29
AU2024266825A1 (en) 2024-12-12
EP3784689A1 (en) 2021-03-03
CN112041334A (zh) 2020-12-04
WO2019210078A1 (en) 2019-10-31
KR20210005146A (ko) 2021-01-13
EP3784689A4 (en) 2022-01-19
AU2019257708A1 (en) 2020-09-03
IL277036A (en) 2020-10-29
JP2021521849A (ja) 2021-08-30
KR102912481B1 (ko) 2026-01-15
JP2026099845A (ja) 2026-06-18

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