JP2024521750A - 塩基エディターの自己不活性化のための組成物及び方法 - Google Patents

塩基エディターの自己不活性化のための組成物及び方法 Download PDF

Info

Publication number
JP2024521750A
JP2024521750A JP2023572544A JP2023572544A JP2024521750A JP 2024521750 A JP2024521750 A JP 2024521750A JP 2023572544 A JP2023572544 A JP 2023572544A JP 2023572544 A JP2023572544 A JP 2023572544A JP 2024521750 A JP2024521750 A JP 2024521750A
Authority
JP
Japan
Prior art keywords
seq
intron
polynucleotide
domain
base editor
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
JP2023572544A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024521750A5 (enExample
Inventor
デビッド ブライソン、
ジャック サリバン、
Original Assignee
ビーム セラピューティクス インク.
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 ビーム セラピューティクス インク. filed Critical ビーム セラピューティクス インク.
Publication of JP2024521750A publication Critical patent/JP2024521750A/ja
Publication of JP2024521750A5 publication Critical patent/JP2024521750A5/ja
Pending legal-status Critical Current

Links

Images

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
    • 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
    • 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
    • C12N15/10—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
    • 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
    • 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
    • C12N15/86—Viral vectors
    • 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
    • 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
    • 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
    • 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)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • 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
    • C12N2310/20—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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (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)
  • Virology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Photoreceptors In Electrophotography (AREA)
  • Dental Preparations (AREA)
JP2023572544A 2021-05-28 2022-05-27 塩基エディターの自己不活性化のための組成物及び方法 Pending JP2024521750A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163194431P 2021-05-28 2021-05-28
US63/194,431 2021-05-28
PCT/US2022/031419 WO2022251687A2 (en) 2021-05-28 2022-05-27 Compositions and methods for the self-inactivation of base editors

Publications (2)

Publication Number Publication Date
JP2024521750A true JP2024521750A (ja) 2024-06-04
JP2024521750A5 JP2024521750A5 (enExample) 2025-06-03

Family

ID=84230360

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023572544A Pending JP2024521750A (ja) 2021-05-28 2022-05-27 塩基エディターの自己不活性化のための組成物及び方法

Country Status (8)

Country Link
US (1) US20240132868A1 (enExample)
EP (1) EP4346840A4 (enExample)
JP (1) JP2024521750A (enExample)
KR (1) KR20240012377A (enExample)
CN (1) CN117729926A (enExample)
AU (1) AU2022280952A1 (enExample)
CA (1) CA3219628A1 (enExample)
WO (1) WO2022251687A2 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118813611B (zh) * 2023-04-19 2025-09-16 清华大学 一种基于rna核酶的dear核酸操纵系统及其应用
AU2024384487A1 (en) * 2023-11-20 2026-06-04 Beam Therapeutics Inc. Compositions and methods for altering complement activation

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018522566A (ja) * 2015-08-07 2018-08-16 カリブー・バイオサイエンシーズ・インコーポレイテッド 操作されたcrispr−cas9組成物及び使用方法
WO2020028823A1 (en) * 2018-08-03 2020-02-06 Beam Therapeutics Inc. Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence
WO2020051561A1 (en) * 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
JP2021500041A (ja) * 2017-10-23 2021-01-07 ストーク セラピューティクス,インク. ナンセンス変異依存rna分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー
WO2021041885A2 (en) * 2019-08-29 2021-03-04 Beam Therapeutics Inc. Compositions and methods for editing a mutation to permit transcription or expression
WO2021050571A1 (en) * 2019-09-09 2021-03-18 Beam Therapeutics Inc. Novel nucleobase editors and methods of using same

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018027078A1 (en) * 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
JP2021505159A (ja) * 2017-12-06 2021-02-18 ジェネレーション バイオ カンパニー 修飾型閉端dna(cedna)を使用する遺伝子編集
KR102853088B1 (ko) * 2018-05-11 2025-09-02 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018522566A (ja) * 2015-08-07 2018-08-16 カリブー・バイオサイエンシーズ・インコーポレイテッド 操作されたcrispr−cas9組成物及び使用方法
JP2021500041A (ja) * 2017-10-23 2021-01-07 ストーク セラピューティクス,インク. ナンセンス変異依存rna分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー
WO2020028823A1 (en) * 2018-08-03 2020-02-06 Beam Therapeutics Inc. Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence
WO2020051561A1 (en) * 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
WO2021041885A2 (en) * 2019-08-29 2021-03-04 Beam Therapeutics Inc. Compositions and methods for editing a mutation to permit transcription or expression
WO2021050571A1 (en) * 2019-09-09 2021-03-18 Beam Therapeutics Inc. Novel nucleobase editors and methods of using same

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NUCLEIC ACID RES., vol. 46, no. 14, JPN6026014898, 2018, pages 84, ISSN: 0005846245 *

Also Published As

Publication number Publication date
WO2022251687A2 (en) 2022-12-01
US20240132868A1 (en) 2024-04-25
AU2022280952A9 (en) 2023-11-30
CA3219628A1 (en) 2022-12-01
WO2022251687A3 (en) 2023-01-19
KR20240012377A (ko) 2024-01-29
AU2022280952A1 (en) 2023-11-23
EP4346840A2 (en) 2024-04-10
CN117729926A (zh) 2024-03-19
EP4346840A4 (en) 2025-06-04

Similar Documents

Publication Publication Date Title
JP7717684B2 (ja) 新規核酸塩基エディター及びその使用方法
JP7646554B2 (ja) アルファ-1アンチトリプシン不全を治療するための組成物および方法
CN114072509A (zh) 脱氨反应脱靶减低的核碱基编辑器和使用其修饰核碱基靶序列的方法
CA3297197A1 (en) Compositions and methods for treating hemoglobinopathies
US20240158775A1 (en) Adenosine deaminase variants and uses thereof
WO2020236936A1 (en) Methods of editing a single nucleotide polymorphism using programmable base editor systems
AU2019266327A1 (en) Methods of editing single nucleotide polymorphism using programmable base editor systems
US20240360433A1 (en) Compositions and methods for the treatment of hereditary angioedema (hae)
US20240117345A1 (en) Compositions and methods for treating transthyretin amyloidosis
CA3198671A1 (en) Compositions and methods for treating glycogen storage disease type 1a
EP3965832A1 (en) Compositions and methods for treating hepatitis b
WO2023049935A1 (en) Compositions and methods for treating hepatitis b virus infection
US20240132868A1 (en) Compositions and methods for the self-inactivation of base editors
WO2023064858A1 (en) Compositions and methods for genome editing the neonatal fc receptor
WO2023115012A2 (en) Compositions and methods for the treatment of spinal muscular atrophy (sma)

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250526

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250526

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20260422

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260721