JP2024503249A - 部位特異的変異導入のための組成物及び方法 - Google Patents

部位特異的変異導入のための組成物及び方法 Download PDF

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JP2024503249A
JP2024503249A JP2023537550A JP2023537550A JP2024503249A JP 2024503249 A JP2024503249 A JP 2024503249A JP 2023537550 A JP2023537550 A JP 2023537550A JP 2023537550 A JP2023537550 A JP 2023537550A JP 2024503249 A JP2024503249 A JP 2024503249A
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cell
deletions
target site
fusion polypeptide
gene
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JP2024503249A5 (https=
JPWO2022140150A5 (https=
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ジョーダン ジャージョル，
アン－レイチェル クロスタグ，
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２セブンティ バイオ インコーポレイテッド
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    • 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
    • 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
    • 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
    • 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
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • 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
    • C12N5/0638—Cytotoxic T lymphocytes [CTL] or lymphokine activated killer cells [LAK]
    • 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
    • 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
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/11—Exodeoxyribonucleases producing 5'-phosphomonoesters (3.1.11)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/11—Exodeoxyribonucleases producing 5'-phosphomonoesters (3.1.11)
    • C12Y301/11001—Exodeoxyribonuclease I (3.1.11.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/11—Exodeoxyribonucleases producing 5'-phosphomonoesters (3.1.11)
    • C12Y301/11002—Exodeoxyribonuclease III (3.1.11.2)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Developmental Biology & Embryology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
JP2023537550A 2020-12-21 2021-12-16 部位特異的変異導入のための組成物及び方法 Pending JP2024503249A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063128391P 2020-12-21 2020-12-21
US63/128,391 2020-12-21
PCT/US2021/063771 WO2022140150A1 (en) 2020-12-21 2021-12-16 Compositions and methods for site-directed mutagenesis

Publications (3)

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JP2024503249A true JP2024503249A (ja) 2024-01-25
JP2024503249A5 JP2024503249A5 (https=) 2024-12-23
JPWO2022140150A5 JPWO2022140150A5 (https=) 2024-12-23

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JP2023537550A Pending JP2024503249A (ja) 2020-12-21 2021-12-16 部位特異的変異導入のための組成物及び方法

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US (1) US20240336904A1 (https=)
EP (1) EP4262850A4 (https=)
JP (1) JP2024503249A (https=)
KR (1) KR20230122628A (https=)
CN (1) CN116887853A (https=)
AU (1) AU2021410632A1 (https=)
CA (1) CA3202889A1 (https=)
WO (1) WO2022140150A1 (https=)

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015516146A (ja) * 2012-02-24 2015-06-11 フレッド ハッチンソン キャンサー リサーチ センター 異常ヘモグロビン症の治療のための組成物および方法
JP2016520320A (ja) * 2013-05-31 2016-07-14 セレクティスＣｅｌｌｅｃｔｉｓ Ｔ細胞受容体α遺伝子を開裂するＬＡＧＬＩＤＡＤＧホーミングエンドヌクレアーゼおよびその用途
JP2018520648A (ja) * 2015-05-13 2018-08-02 シアトル チルドレンズ ホスピタル， ディービーエー シアトル チルドレンズ リサーチ インスティテュート 初代細胞におけるエンドヌクレアーゼに基づいた遺伝子編集の向上
JP2019509738A (ja) * 2016-03-11 2019-04-11 ブルーバード バイオ， インコーポレイテッド ゲノム編集された免疫エフェクター細胞
WO2019186275A1 (en) * 2018-03-27 2019-10-03 G+Flas Life Sciences Sequence-specific in vivo cell targeting
JP2019532674A (ja) * 2016-10-11 2019-11-14 ブルーバード バイオ， インコーポレイテッド ＴＣＲαホーミングエンドヌクレアーゼバリアント
US20200109385A1 (en) * 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
WO2020123371A2 (en) * 2018-12-10 2020-06-18 Bluebird Bio, Inc. Homing endonuclease variants
WO2020252110A1 (en) * 2019-06-14 2020-12-17 Bluebird Bio, Inc. Compositions and methods for treating cancer

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012118717A2 (en) * 2011-02-28 2012-09-07 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drive high efficiency gene disruption
IL313037A (en) * 2017-05-25 2024-07-01 2Seventy Bio Inc Cblb endonuclease variants, compositions, and methods of use

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015516146A (ja) * 2012-02-24 2015-06-11 フレッド ハッチンソン キャンサー リサーチ センター 異常ヘモグロビン症の治療のための組成物および方法
JP2016520320A (ja) * 2013-05-31 2016-07-14 セレクティスＣｅｌｌｅｃｔｉｓ Ｔ細胞受容体α遺伝子を開裂するＬＡＧＬＩＤＡＤＧホーミングエンドヌクレアーゼおよびその用途
JP2018520648A (ja) * 2015-05-13 2018-08-02 シアトル チルドレンズ ホスピタル， ディービーエー シアトル チルドレンズ リサーチ インスティテュート 初代細胞におけるエンドヌクレアーゼに基づいた遺伝子編集の向上
JP2019509738A (ja) * 2016-03-11 2019-04-11 ブルーバード バイオ， インコーポレイテッド ゲノム編集された免疫エフェクター細胞
JP2019532674A (ja) * 2016-10-11 2019-11-14 ブルーバード バイオ， インコーポレイテッド ＴＣＲαホーミングエンドヌクレアーゼバリアント
WO2019186275A1 (en) * 2018-03-27 2019-10-03 G+Flas Life Sciences Sequence-specific in vivo cell targeting
US20200109385A1 (en) * 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
WO2020123371A2 (en) * 2018-12-10 2020-06-18 Bluebird Bio, Inc. Homing endonuclease variants
WO2020252110A1 (en) * 2019-06-14 2020-12-17 Bluebird Bio, Inc. Compositions and methods for treating cancer

Also Published As

Publication number Publication date
KR20230122628A (ko) 2023-08-22
WO2022140150A1 (en) 2022-06-30
EP4262850A4 (en) 2025-07-09
EP4262850A1 (en) 2023-10-25
AU2021410632A1 (en) 2023-08-03
CA3202889A1 (en) 2022-06-30
CN116887853A (zh) 2023-10-13
US20240336904A1 (en) 2024-10-10

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