JP7646552B2 - 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ - Google Patents

低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ Download PDF

Info

Publication number
JP7646552B2
JP7646552B2 JP2021544294A JP2021544294A JP7646552B2 JP 7646552 B2 JP7646552 B2 JP 7646552B2 JP 2021544294 A JP2021544294 A JP 2021544294A JP 2021544294 A JP2021544294 A JP 2021544294A JP 7646552 B2 JP7646552 B2 JP 7646552B2
Authority
JP
Japan
Prior art keywords
deaminase
cas9
amino acid
fusion protein
domain
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.)
Active
Application number
JP2021544294A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022519507A5 (https=
JP2022519507A (ja
JPWO2020160514A5 (https=
Inventor
イアン スレイメイカー、
ジェイソン マイケル ゲールケ、
ニコル ゴーデッリ、
イ ユ、
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 JP2022519507A publication Critical patent/JP2022519507A/ja
Publication of JP2022519507A5 publication Critical patent/JP2022519507A5/ja
Publication of JPWO2020160514A5 publication Critical patent/JPWO2020160514A5/ja
Priority to JP2025034660A priority Critical patent/JP2025102784A/ja
Application granted granted Critical
Publication of JP7646552B2 publication Critical patent/JP7646552B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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
    • C12N15/10—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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • 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)
    • C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/04—Libraries containing only organic compounds
    • C40B40/10—Libraries containing peptides or polypeptides, or derivatives thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021544294A 2019-01-31 2020-01-31 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ Active JP7646552B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025034660A JP2025102784A (ja) 2019-01-31 2025-03-05 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962799702P 2019-01-31 2019-01-31
US62/799,702 2019-01-31
PCT/US2020/016285 WO2020160514A1 (en) 2019-01-31 2020-01-31 Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025034660A Division JP2025102784A (ja) 2019-01-31 2025-03-05 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ

Publications (4)

Publication Number Publication Date
JP2022519507A JP2022519507A (ja) 2022-03-24
JP2022519507A5 JP2022519507A5 (https=) 2023-02-06
JPWO2020160514A5 JPWO2020160514A5 (https=) 2023-02-06
JP7646552B2 true JP7646552B2 (ja) 2025-03-17

Family

ID=71842366

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021544294A Active JP7646552B2 (ja) 2019-01-31 2020-01-31 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ
JP2025034660A Pending JP2025102784A (ja) 2019-01-31 2025-03-05 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025034660A Pending JP2025102784A (ja) 2019-01-31 2025-03-05 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ

Country Status (7)

Country Link
EP (1) EP3918078A4 (https=)
JP (2) JP7646552B2 (https=)
KR (1) KR20210121113A (https=)
CN (1) CN114040977B (https=)
AU (1) AU2020215730A1 (https=)
CA (1) CA3127493A1 (https=)
WO (1) WO2020160514A1 (https=)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
IL296024A (en) 2020-03-04 2022-10-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for modulating a genome
WO2021222318A1 (en) 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene
CA3194019A1 (en) * 2020-09-04 2022-03-10 National University Corporation Kobe University Miniaturized cytidine deaminase-containing complex for modifying double-stranded dna
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CN116601293A (zh) * 2020-12-07 2023-08-15 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法
CN113308451B (zh) * 2020-12-07 2023-07-25 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法
CA3214494A1 (en) * 2021-03-26 2022-09-29 Beam Therapeutics Inc. Adenosine deaminase variants and uses thereof
WO2022221699A1 (en) * 2021-04-16 2022-10-20 Beam Therapeutics, Inc. Genetic modification of hepatocytes
WO2023283092A1 (en) * 2021-07-06 2023-01-12 Prime Medicine, Inc. Compositions and methods for efficient genome editing
JP2024533311A (ja) 2021-09-08 2024-09-12 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー ゲノムを調節するための方法及び組成物
CN117925585A (zh) * 2022-04-07 2024-04-26 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
WO2025119307A1 (zh) * 2023-12-06 2025-06-12 北京齐禾生科生物科技有限公司 优化的qbe碱基编辑系统及其应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017048969A1 (en) 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018213726A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2019005884A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-ADENINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED NUCLEIC ACID EDITION

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CA2971444A1 (en) * 2014-12-20 2016-06-23 Arc Bio, Llc Compositions and methods for targeted depletion, enrichment, and partitioning of nucleic acids using crispr/cas system proteins
TWI813532B (zh) * 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的ｃｒｉｓｐｒ酶突變
JP7109784B2 (ja) * 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
WO2018119359A1 (en) * 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017048969A1 (en) 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018213726A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2019005884A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-ADENINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED NUCLEIC ACID EDITION

Also Published As

Publication number Publication date
EP3918078A1 (en) 2021-12-08
WO2020160514A1 (en) 2020-08-06
EP3918078A4 (en) 2022-12-14
CN114040977B (zh) 2024-09-03
CA3127493A1 (en) 2020-08-06
JP2025102784A (ja) 2025-07-08
CN114040977A (zh) 2022-02-11
KR20210121113A (ko) 2021-10-07
JP2022519507A (ja) 2022-03-24
AU2020215730A1 (en) 2021-07-29
US20220098572A1 (en) 2022-03-31

Similar Documents

Publication Publication Date Title
JP7646552B2 (ja) 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ
JP7657711B2 (ja) 核酸塩基編集システムを送達するための組成物および方法
JP7717684B2 (ja) 新規核酸塩基エディター及びその使用方法
JP7679311B2 (ja) プログラム可能塩基エディターシステムを用いた一塩基多型編集法
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
CN114026237B (zh) 用于治疗1a型糖原贮积病的组成物和方法
WO2020168133A1 (en) Compositions and methods for treating hemoglobinopathies
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
KR20230158476A (ko) 유전자 요법을 위한 재조합 광견병 바이러스
CN114929287A (zh) 用于治疗乙型肝炎的组合物和方法
AU2021360919A1 (en) Compositions and methods for treating glycogen storage disease type 1a
JP2025533556A (ja) 合成ポリペプチド及びその用途
US12612618B2 (en) Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230127

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230127

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231220

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240321

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240702

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241001

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250204

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250305

R150 Certificate of patent or registration of utility model

Ref document number: 7646552

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150