JP2024502348A - Ｔｒａｃｒ不含 Ｖ型Ｃａｓシステム用のガイドＲＮＡデザインおよび複合体 - Google Patents

Ｔｒａｃｒ不含 Ｖ型Ｃａｓシステム用のガイドＲＮＡデザインおよび複合体 Download PDF

Info

Publication number
JP2024502348A
JP2024502348A JP2023541024A JP2023541024A JP2024502348A JP 2024502348 A JP2024502348 A JP 2024502348A JP 2023541024 A JP2023541024 A JP 2023541024A JP 2023541024 A JP2023541024 A JP 2023541024A JP 2024502348 A JP2024502348 A JP 2024502348A
Authority
JP
Japan
Prior art keywords
grna
ligand binding
complex
linker
binding complex
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2023541024A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024502348A5 (https=
Inventor
ダニエル マーシャル、カート
ブエノ マチャド、ハイド
アンダーソン、エミリー
ヘイル、アレクサンダー
スミス、アマンダ
ヘンプヒル、ケビン
コーフマン、アナスタシア
ナンティ、リー
ダニエル ラシュトン、マイケル
Original Assignee
ホライズン・ディスカバリー・リミテッド
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 ホライズン・ディスカバリー・リミテッド filed Critical ホライズン・ディスカバリー・リミテッド
Publication of JP2024502348A publication Critical patent/JP2024502348A/ja
Publication of JP2024502348A5 publication Critical patent/JP2024502348A5/ja
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N5/0684—Cells of the urinary tract or kidneys
    • C12N5/0686—Kidney 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
    • 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]
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • 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
    • C12N2310/16—Aptamers
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
JP2023541024A 2021-01-05 2022-01-05 Ｔｒａｃｒ不含 Ｖ型Ｃａｓシステム用のガイドＲＮＡデザインおよび複合体 Pending JP2024502348A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163133942P 2021-01-05 2021-01-05
US63/133,942 2021-01-05
PCT/US2022/011289 WO2022150367A1 (en) 2021-01-05 2022-01-05 Guide rna designs and complexes for tracr-less type v cas systems

Publications (2)

Publication Number Publication Date
JP2024502348A true JP2024502348A (ja) 2024-01-18
JP2024502348A5 JP2024502348A5 (https=) 2025-01-10

Family

ID=82358156

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023541024A Pending JP2024502348A (ja) 2021-01-05 2022-01-05 Ｔｒａｃｒ不含 Ｖ型Ｃａｓシステム用のガイドＲＮＡデザインおよび複合体

Country Status (6)

Country Link
US (1) US20240067983A1 (https=)
EP (1) EP4274902A4 (https=)
JP (1) JP2024502348A (https=)
CN (1) CN117222743A (https=)
CA (1) CA3207138A1 (https=)
WO (1) WO2022150367A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
AU2020215730A1 (en) 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors
CN120174005A (zh) 2019-02-13 2025-06-20 比姆医疗股份有限公司 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
WO2025212120A1 (en) * 2024-04-05 2025-10-09 Arbor Biotechnologies, Inc. Chemical modifications of guide rnas for crispr nucleases

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018035388A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2019217942A1 (en) * 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7044373B2 (ja) * 2015-07-15 2022-03-30 ラトガース，ザ ステート ユニバーシティ オブ ニュージャージー ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
MX2021010559A (es) * 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
US12286633B2 (en) * 2019-12-17 2025-04-29 University Of Maryland, College Park Compositions and methods for genome editing in plants

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018035388A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2019217942A1 (en) * 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
COLLANTES JC. ET AL., CRISPR J, vol. 4, JPN6026003709, February 2021 (2021-02-01), pages 58 - 68, ISSN: 0005787693 *
LI C. ET AL., GENOME BIOL, vol. Vol. 21:141, JPN6026003707, 2020, ISSN: 0005787692 *
LI T. ET AL., BIOTECHNOL ADV, vol. 37, JPN6026003710, 2019, pages 21 - 27, ISSN: 0005787694 *

Also Published As

Publication number Publication date
US20240067983A1 (en) 2024-02-29
CN117222743A (zh) 2023-12-12
WO2022150367A1 (en) 2022-07-14
EP4274902A1 (en) 2023-11-15
EP4274902A4 (en) 2025-04-16
CA3207138A1 (en) 2022-07-14

Similar Documents

Publication Publication Date Title
US20240060088A1 (en) Guide RNA Designs and Complexes for Type V Cas Systems
CN115651927B (zh) 编辑rna的方法和组合物
AU2021282533C1 (en) Optimized crispr/cas9 systems and methods for gene editing in stem cells
JP2024502348A (ja) Ｔｒａｃｒ不含 Ｖ型Ｃａｓシステム用のガイドＲＮＡデザインおよび複合体
JP7101419B2 (ja) 内因性ｔ細胞受容体の標的置換
JP2023123561A (ja) 移植を改善するためのｃｒｉｓｐｒ／ｃａｓ関連方法および組成物
CA2968336C (en) Construct for site directed editing of an adenosine nucleotide in target rna
CN113939591A (zh) 编辑rna的方法和组合物
JP2022550599A (ja) 操作されたデュアルガイド核酸を有するｃｒｉｓｐｒシステム
KR20170106486A (ko) 일차 조혈 세포에서의 단백질 전달
WO2015148670A1 (en) Crispr/cas-related methods and compositions for treating hiv infection and aids
EP4419672A2 (en) Compositions and methods for targeting, editing, or modifying genes
CN116507629B (zh) Rna支架
US20240309347A1 (en) Fusion Proteins for CRISPR-based Transcriptional Repression
US20220251566A1 (en) Cells engineered for oligonucleotide delivery, and methods for making and using thereof
HK40081918B (en) Methods and compositions for editing rna
WO2026019837A1 (en) Compositions and methods for overcoming t-cell exhaustion
HK40081918A (en) Methods and compositions for editing rna
HK40061041A (en) Methods and compositions for editing rnas
HK40056042A (en) Methods and compositions for editing rnas
HK40056042B (en) Methods and compositions for editing rnas

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241224

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20241224

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241225

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20260203

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260428