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
JPWO2020160514A5 (cg-RX-API-DMAC7.html
JP2022519507A (ja
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 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

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
    • CCHEMISTRY; METALLURGY
    • C40COMBINATORIAL TECHNOLOGY
    • C40BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00Libraries per se, e.g. arrays, mixtures
    • C40B40/04Libraries containing only organic compounds
    • C40B40/10Libraries containing peptides or polypeptides, or derivatives thereof
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005Cytidine 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)
  • Wood Science & Technology (AREA)
  • Zoology (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)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (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 (3)

Publication Number Publication Date
JP2022519507A JP2022519507A (ja) 2022-03-24
JPWO2020160514A5 JPWO2020160514A5 (cg-RX-API-DMAC7.html) 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 (8)

Country Link
US (1) US20220098572A1 (cg-RX-API-DMAC7.html)
EP (1) EP3918078A4 (cg-RX-API-DMAC7.html)
JP (2) JP7646552B2 (cg-RX-API-DMAC7.html)
KR (1) KR20210121113A (cg-RX-API-DMAC7.html)
CN (1) CN114040977B (cg-RX-API-DMAC7.html)
AU (1) AU2020215730A1 (cg-RX-API-DMAC7.html)
CA (1) CA3127493A1 (cg-RX-API-DMAC7.html)
WO (1) WO2020160514A1 (cg-RX-API-DMAC7.html)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
CA3100019A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
AU2021230546A1 (en) 2020-03-04 2022-10-13 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
US20230159913A1 (en) 2020-04-28 2023-05-25 The Broad Institute, Inc. Targeted base editing of the ush2a gene
KR20230061474A (ko) * 2020-09-04 2023-05-08 고쿠리츠다이가쿠호진 고베다이가쿠 소형화 시티딘 데아미나아제를 포함하는 이중쇄 dna의 개변용 복합체
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
CN112195164B (zh) * 2020-12-07 2021-04-23 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
CA3224970A1 (en) * 2021-07-06 2023-01-12 Holly A. REES Compositions and methods for efficient genome editing
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
US20250361530A1 (en) * 2022-05-14 2025-11-27 The Regents Of The University Of California Genome editing systems for multiplexing point mutation introduction in living cells
CN119591727B (zh) * 2023-12-06 2025-09-30 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 (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150166982A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
WO2015089486A2 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
CN114438169B (zh) * 2014-12-20 2024-11-08 阿克生物公司 使用CRISPR/Cas系统蛋白靶向消减、富集、和分割核酸的组合物及方法
EP3250689B1 (en) * 2015-01-28 2020-11-04 The Regents of The University of California Methods and compositions for labeling a single-stranded target nucleic acid
EP3303634B1 (en) * 2015-06-03 2023-08-30 The Regents of The University of California Cas9 variants and methods of use thereof
WO2016205613A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
SG11201803173VA (en) * 2015-10-23 2018-05-30 Harvard College Nucleobase editors and uses thereof
JP7231935B2 (ja) * 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
SG10201913505WA (en) * 2016-10-17 2020-02-27 Univ Nanyang Tech Truncated crispr-cas proteins for dna targeting
US10745677B2 (en) * 2016-12-23 2020-08-18 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
JP2025102784A (ja) 2025-07-08
CA3127493A1 (en) 2020-08-06
EP3918078A4 (en) 2022-12-14
KR20210121113A (ko) 2021-10-07
CN114040977A (zh) 2022-02-11
JP2022519507A (ja) 2022-03-24
US20220098572A1 (en) 2022-03-31
CN114040977B (zh) 2024-09-03
EP3918078A1 (en) 2021-12-08
AU2020215730A1 (en) 2021-07-29
WO2020160514A1 (en) 2020-08-06

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
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
EP3924481A1 (en) Compositions and methods for treating hemoglobinopathies
CN114026237B (zh) 用于治疗1a型糖原贮积病的组成物和方法
CN114929287A (zh) 用于治疗乙型肝炎的组合物和方法
KR20230158476A (ko) 유전자 요법을 위한 재조합 광견병 바이러스
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

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