JP2024531217A - 遺伝子改変高忠実度ｏｍｎｉ－５０ヌクレアーゼバリアント - Google Patents

遺伝子改変高忠実度ｏｍｎｉ－５０ヌクレアーゼバリアント Download PDF

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JP2024531217A
JP2024531217A JP2024508545A JP2024508545A JP2024531217A JP 2024531217 A JP2024531217 A JP 2024531217A JP 2024508545 A JP2024508545 A JP 2024508545A JP 2024508545 A JP2024508545 A JP 2024508545A JP 2024531217 A JP2024531217 A JP 2024531217A
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omni
amino acid
nuclease
cells
nuclease variant
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JPWO2023019263A5 (https=
JP2024531217A5 (https=
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イザール、リオル
エマニュエル、ラフィ
ロッカー、リアット
ヘルマン、アサエル
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エメンドバイオ・インコーポレイテッド
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Publication of JPWO2023019263A5 publication Critical patent/JPWO2023019263A5/ja
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    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024508545A 2021-08-12 2022-08-12 遺伝子改変高忠実度ｏｍｎｉ－５０ヌクレアーゼバリアント Pending JP2024531217A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163232571P 2021-08-12 2021-08-12
US63/232,571 2021-08-12
US202263332214P 2022-04-18 2022-04-18
US63/332,214 2022-04-18
US202263333037P 2022-04-20 2022-04-20
US63/333,037 2022-04-20
PCT/US2022/074930 WO2023019263A1 (en) 2021-08-12 2022-08-12 Engineered high fidelity omni-50 nuclease variants

Publications (3)

Publication Number Publication Date
JP2024531217A true JP2024531217A (ja) 2024-08-29
JPWO2023019263A5 JPWO2023019263A5 (https=) 2025-08-20
JP2024531217A5 JP2024531217A5 (https=) 2025-08-20

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ID=85201002

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JP2024508545A Pending JP2024531217A (ja) 2021-08-12 2022-08-12 遺伝子改変高忠実度ｏｍｎｉ－５０ヌクレアーゼバリアント

Country Status (8)

Country Link
US (1) US20250283115A1 (https=)
EP (1) EP4384609A4 (https=)
JP (1) JP2024531217A (https=)
KR (1) KR20240043792A (https=)
AU (1) AU2022326575A1 (https=)
CA (1) CA3228366A1 (https=)
IL (1) IL310730A (https=)
WO (1) WO2023019263A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12091688B2 (en) 2021-02-08 2024-09-17 Emendobio Inc. OMNI-103 CRISPR nuclease
KR20230142740A (ko) 2021-02-08 2023-10-11 에멘도바이오 인코포레이티드 Omni-103 crispr 뉴클레아제
CN119546757A (zh) 2022-05-25 2025-02-28 宏基因组学公司 肝酶表达的补充
US20260021141A1 (en) * 2022-07-15 2026-01-22 Emendobio Inc. Strategies For Knock-Ins At APLP2 Safe Harbor Sites
WO2025117858A1 (en) * 2023-11-28 2025-06-05 Metagenomi, Inc. Supplementation of liver enzyme expression

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9512446B1 (en) * 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
WO2020223514A2 (en) * 2019-04-30 2020-11-05 Emendobio Inc. Novel omni-50 crispr nuclease
WO2020223553A2 (en) * 2019-04-30 2020-11-05 Emendobio Inc. Novel omni crispr nucleases
US12435334B2 (en) * 2019-11-06 2025-10-07 Emendobio Inc. Differential knockout of an allele of a heterozygous ELANE gene-II
WO2024168159A1 (en) * 2023-02-08 2024-08-15 Emendobio Inc. Engineered omni-50 nuclease variants

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Publication number Publication date
KR20240043792A (ko) 2024-04-03
AU2022326575A1 (en) 2024-03-14
IL310730A (en) 2024-04-01
WO2023019263A1 (en) 2023-02-16
EP4384609A1 (en) 2024-06-19
CA3228366A1 (en) 2023-02-16
EP4384609A4 (en) 2025-07-02
US20250283115A1 (en) 2025-09-11

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