AU2020215730A1 - Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors - Google Patents

Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors Download PDF

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Publication number
AU2020215730A1
AU2020215730A1 AU2020215730A AU2020215730A AU2020215730A1 AU 2020215730 A1 AU2020215730 A1 AU 2020215730A1 AU 2020215730 A AU2020215730 A AU 2020215730A AU 2020215730 A AU2020215730 A AU 2020215730A AU 2020215730 A1 AU2020215730 A1 AU 2020215730A1
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AU
Australia
Prior art keywords
deaminase
fusion protein
cas9
amino acid
terminal fragment
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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
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AU2020215730A
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English (en)
Inventor
Nicole GAUDELLI
Jason Michael GEHRKE
Ian SLAYMAKER
Yi Yu
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Beam Therapeutics Inc
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Beam Therapeutics Inc
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Publication of AU2020215730A1 publication Critical patent/AU2020215730A1/en
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    • 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
    • C12N15/11—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
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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
    • C12N15/11—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)

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  • 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)
  • 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)
AU2020215730A 2019-01-31 2020-01-31 Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors Pending AU2020215730A1 (en)

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

Publications (1)

Publication Number Publication Date
AU2020215730A1 true AU2020215730A1 (en) 2021-07-29

Family

ID=71842366

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020215730A Pending AU2020215730A1 (en) 2019-01-31 2020-01-31 Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors

Country Status (8)

Country Link
US (1) US20220098572A1 (enExample)
EP (1) EP3918078A4 (enExample)
JP (2) JP7646552B2 (enExample)
KR (1) KR20210121113A (enExample)
CN (1) CN114040977B (enExample)
AU (1) AU2020215730A1 (enExample)
CA (1) CA3127493A1 (enExample)
WO (1) WO2020160514A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
JP7642531B2 (ja) 2018-05-11 2025-03-10 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
MX2022010991A (es) 2020-03-04 2023-02-09 Flagship Pioneering Innovations Vi Llc Metodos y composiciones para modular un genoma.
US20230159913A1 (en) 2020-04-28 2023-05-25 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
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
AU2022306377A1 (en) * 2021-07-06 2024-01-25 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
WO2023225451A1 (en) * 2022-05-14 2023-11-23 The Regents Of The University Of California Genome editing systems for multiplexing point mutation introduction in living cells
CN119592626A (zh) * 2023-12-06 2025-03-11 北京齐禾生科生物科技有限公司 优化工程化t细胞的碱基编辑系统及其应用

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US9840699B2 (en) * 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
DK3234200T3 (da) * 2014-12-20 2021-08-23 Arc Bio Llc Fremgangsmåde til målrettet depletering af nukleinsyrer ved anvendelse af CRISPR/CAS-systemproteiner
US11180792B2 (en) * 2015-01-28 2021-11-23 The Regents Of The University Of California Methods and compositions for labeling a single-stranded target nucleic acid
WO2016196655A1 (en) * 2015-06-03 2016-12-08 The Regents Of The University Of California Cas9 variants and methods of use thereof
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
EP3350203A4 (en) * 2015-09-17 2019-06-26 The Regents of The University of California POLYPEPTIDES OF CAS9 VARIANT WITH INTERNAL INSERTIONS
IL258821B (en) * 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
KR20250103795A (ko) * 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
SG10201913505WA (en) * 2016-10-17 2020-02-27 Univ Nanyang Tech Truncated crispr-cas proteins for dna targeting
WO2018119359A1 (en) * 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
BR112019019655A2 (pt) * 2017-03-23 2020-04-22 Harvard College editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CA3064601A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. Crispr/cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing

Also Published As

Publication number Publication date
EP3918078A4 (en) 2022-12-14
JP2025102784A (ja) 2025-07-08
JP2022519507A (ja) 2022-03-24
EP3918078A1 (en) 2021-12-08
CN114040977A (zh) 2022-02-11
US20220098572A1 (en) 2022-03-31
CN114040977B (zh) 2024-09-03
CA3127493A1 (en) 2020-08-06
WO2020160514A1 (en) 2020-08-06
JP7646552B2 (ja) 2025-03-17
KR20210121113A (ko) 2021-10-07

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