CA3192224A1 - Base editing enzymes - Google Patents

Base editing enzymes

Info

Publication number
CA3192224A1
CA3192224A1 CA3192224A CA3192224A CA3192224A1 CA 3192224 A1 CA3192224 A1 CA 3192224A1 CA 3192224 A CA3192224 A CA 3192224A CA 3192224 A CA3192224 A CA 3192224A CA 3192224 A1 CA3192224 A1 CA 3192224A1
Authority
CA
Canada
Prior art keywords
endonuclease
nucleic acid
sequence
seq
nos
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
CA3192224A
Other languages
English (en)
French (fr)
Inventor
Jyun-Liang LIN
Alan Brooks
Cristina Butterfield
Christopher Brown
Cindy CASTELLE
Brian C. Thomas
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Metagenomi Therapeutics Inc
Original Assignee
Metagenomi Inc
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 Metagenomi Inc filed Critical Metagenomi Inc
Publication of CA3192224A1 publication Critical patent/CA3192224A1/en
Pending legal-status Critical Current

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    • 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
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    • 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
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    • 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
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    • 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
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/70Vectors or expression systems specially adapted for E. coli
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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
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    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • 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]
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2497Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing N- glycosyl compounds (3.2.2)
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    • 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)
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    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/02Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2) hydrolysing N-glycosyl compounds (3.2.2)
    • C12Y302/02027Uracil-DNA glycosylase (3.2.2.27)
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    • 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)
    • C12Y305/04004Adenosine deaminase (3.5.4.4)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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    • 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]
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Dental Preparations (AREA)
  • Paints Or Removers (AREA)
  • Graft Or Block Polymers (AREA)
CA3192224A 2020-09-11 2021-09-10 Base editing enzymes Pending CA3192224A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063077057P 2020-09-11 2020-09-11
US63/077,057 2020-09-11
US202163222351P 2021-07-15 2021-07-15
US63/222,351 2021-07-15
PCT/US2021/049962 WO2022056324A1 (en) 2020-09-11 2021-09-10 Base editing enzymes

Publications (1)

Publication Number Publication Date
CA3192224A1 true CA3192224A1 (en) 2022-03-17

Family

ID=80629930

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3192224A Pending CA3192224A1 (en) 2020-09-11 2021-09-10 Base editing enzymes

Country Status (9)

Country Link
US (3) US12286654B2 (enExample)
EP (1) EP4211234A4 (enExample)
JP (1) JP2023540797A (enExample)
KR (1) KR20230062873A (enExample)
AU (1) AU2021339819A1 (enExample)
CA (1) CA3192224A1 (enExample)
GB (1) GB2605340B (enExample)
MX (1) MX2023002913A (enExample)
WO (1) WO2022056324A1 (enExample)

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WO2021178933A2 (en) 2020-03-06 2021-09-10 Metagenomi Ip Technologies, Llc Class ii, type v crispr systems
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
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
WO2021202568A1 (en) * 2020-03-31 2021-10-07 Metagenomi Ip Technologies, Llc Class ii, type ii crispr systems
JP2023540797A (ja) 2020-09-11 2023-09-26 メタゲノミ,インコーポレイテッド 塩基編集酵素
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CA3205865A1 (en) * 2021-01-22 2022-07-28 Brian C. Thomas Novel engineered and chimeric nucleases
AU2022335499A1 (en) 2021-08-27 2024-02-22 Metagenomi Therapeutics, Inc. Enzymes with ruvc domains
WO2023081855A1 (en) * 2021-11-05 2023-05-11 Metagenomi, Inc. Base editing enzymes
AU2022396533A1 (en) 2021-11-24 2024-05-02 Metagenomi Therapeutics, Inc. Endonuclease systems
CN121241133A (zh) * 2023-03-31 2025-12-30 阿伯生物技术公司 Crispr核酸酶多肽和包含此类crispr核酸酶多肽的基因编辑系统
WO2025003344A1 (en) * 2023-06-28 2025-01-02 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2025106894A1 (en) * 2023-11-15 2025-05-22 Profluent Bio Inc. Adenine deaminases and compositions, systems, and methods thereof
WO2025240372A1 (en) * 2024-05-13 2025-11-20 Metagenomi, Inc. Compositions and methods for editing t cells

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CN110214183A (zh) * 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
US11192929B2 (en) 2016-12-08 2021-12-07 Regents Of The University Of Minnesota Site-specific DNA base editing using modified APOBEC enzymes
EP3572525A4 (en) 2017-01-17 2020-09-30 Institute for Basic Science PROCEDURE FOR IDENTIFYING AN OFF-TARGET BASIC EDITING SITE BY BREAKING A SINGLE DNA STRAND
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
EP3617311B1 (en) * 2017-03-30 2024-11-20 Kyoto University Method for inducing exon skipping by genome editing
US9982279B1 (en) * 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
KR102761791B1 (ko) * 2017-06-26 2025-02-05 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법
JP2021506251A (ja) * 2017-12-14 2021-02-22 クリスパー セラピューティクス アーゲー 新規rnaプログラム可能エンドヌクレアーゼ系、ならびにゲノム編集および他の適用におけるその使用
US11946040B2 (en) * 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
US20220298494A1 (en) 2019-02-14 2022-09-22 Metagenomi Ip Technologies, Llc Enzymes with ruvc domains
CA3241703C (en) 2019-02-14 2026-02-24 Metagenomi Therapeutics, Inc. Enzymes with ruvc domains
WO2021097118A1 (en) 2019-11-12 2021-05-20 The Broad Institute, Inc. Small type ii cas proteins and methods of use thereof
WO2021202568A1 (en) * 2020-03-31 2021-10-07 Metagenomi Ip Technologies, Llc Class ii, type ii crispr systems
GB2700205B (en) 2020-09-11 2026-04-15 Metagenomi Therapeutics Inc Base editing enzymes
JP2023540797A (ja) 2020-09-11 2023-09-26 メタゲノミ,インコーポレイテッド 塩基編集酵素
WO2023081855A1 (en) * 2021-11-05 2023-05-11 Metagenomi, Inc. Base editing enzymes

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Publication number Publication date
WO2022056324A1 (en) 2022-03-17
US20230348876A1 (en) 2023-11-02
GB2605340A8 (en) 2024-02-21
GB2605340A (en) 2022-09-28
AU2021339819A1 (en) 2023-05-25
KR20230062873A (ko) 2023-05-09
MX2023002913A (es) 2023-06-01
US20230374482A1 (en) 2023-11-23
EP4211234A4 (en) 2025-04-30
GB2605340B (en) 2024-12-18
JP2023540797A (ja) 2023-09-26
EP4211234A1 (en) 2023-07-19
GB202209534D0 (en) 2022-08-10
US20220364067A1 (en) 2022-11-17
US12286654B2 (en) 2025-04-29

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