JP2024545144A - Omni-103 crisprヌクレアーゼ-rna複合体 - Google Patents

Omni-103 crisprヌクレアーゼ-rna複合体 Download PDF

Info

Publication number
JP2024545144A
JP2024545144A JP2024534340A JP2024534340A JP2024545144A JP 2024545144 A JP2024545144 A JP 2024545144A JP 2024534340 A JP2024534340 A JP 2024534340A JP 2024534340 A JP2024534340 A JP 2024534340A JP 2024545144 A JP2024545144 A JP 2024545144A
Authority
JP
Japan
Prior art keywords
sequence
tracrrna
composition according
seq
nucleotides
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
JP2024534340A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024545144A5 (enExample
Inventor
イザール、リオル
ロッカー、リアット
マールバッハ・バール、ナダブ
ヘヒト、ニル
Original Assignee
エメンドバイオ・インコーポレイテッド
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 エメンドバイオ・インコーポレイテッド filed Critical エメンドバイオ・インコーポレイテッド
Publication of JP2024545144A publication Critical patent/JP2024545144A/ja
Publication of JP2024545144A5 publication Critical patent/JP2024545144A5/ja
Pending legal-status Critical Current

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
    • 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
    • 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
    • C12N15/87Introduction 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
    • 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
    • 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
    • C12N15/113Non-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
    • C12N15/1138Non-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 receptors or cell surface 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
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2024534340A 2021-12-07 2022-12-06 Omni-103 crisprヌクレアーゼ-rna複合体 Pending JP2024545144A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163286855P 2021-12-07 2021-12-07
US63/286,855 2021-12-07
PCT/US2022/081019 WO2023107946A2 (en) 2021-12-07 2022-12-06 Omni-103 crispr nuclease-rna complexes

Publications (2)

Publication Number Publication Date
JP2024545144A true JP2024545144A (ja) 2024-12-05
JP2024545144A5 JP2024545144A5 (enExample) 2025-12-15

Family

ID=86731282

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024534340A Pending JP2024545144A (ja) 2021-12-07 2022-12-06 Omni-103 crisprヌクレアーゼ-rna複合体

Country Status (9)

Country Link
US (1) US20250034596A1 (enExample)
EP (1) EP4444876A2 (enExample)
JP (1) JP2024545144A (enExample)
KR (1) KR20240135610A (enExample)
CN (2) CN116887854A (enExample)
AU (1) AU2022405520A1 (enExample)
CA (1) CA3240329A1 (enExample)
IL (1) IL313397A (enExample)
WO (1) WO2023107946A2 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024506608A (ja) 2021-02-08 2024-02-14 エメンドバイオ・インコーポレイテッド Omni-103 crisprヌクレアーゼ
US12091688B2 (en) 2021-02-08 2024-09-17 Emendobio Inc. OMNI-103 CRISPR nuclease
CN120837655A (zh) * 2025-09-24 2025-10-28 中国人民解放军军事科学院军事医学研究院 磷酸化抑制工具在制备预防和/或治疗三阴性乳腺癌的药物中的用途

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020069029A1 (en) * 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
IL298577A (en) * 2020-05-27 2023-01-01 Emendobio Inc Biallelic silencing of sarm1

Also Published As

Publication number Publication date
KR20240135610A (ko) 2024-09-11
WO2023107946A3 (en) 2023-08-03
IL313397A (en) 2024-08-01
CN118660967A (zh) 2024-09-17
EP4444876A2 (en) 2024-10-16
US20250034596A1 (en) 2025-01-30
CN116887854A (zh) 2023-10-13
WO2023107946A2 (en) 2023-06-15
AU2022405520A1 (en) 2024-07-04
CA3240329A1 (en) 2023-06-15

Similar Documents

Publication Publication Date Title
JP2024050582A (ja) 新規のomni-50crisprヌクレアーゼ
JP2023531384A (ja) 新規なomni-59、61、67、76、79、80、81及び82クリスパーヌクレアーゼ
JP2024545144A (ja) Omni-103 crisprヌクレアーゼ-rna複合体
JP2024516161A (ja) 新規なomni-117、140、150~158、160~165、167~177、180~188、191~198、200、201、203、205~209、211~217、219、220、222、223、226、227、229、231~236、238~245、247、250、254、256、257、260及び262 crisprヌクレアーゼ
JP2023549139A (ja) 新規omni-50 crisprヌクレアーゼ-rna複合体
JP2024532784A (ja) 新規なomni-115、124、127、144~149、159、218、237、248、251~253及び259crisprヌクレアーゼ
JP2024542883A (ja) 改変された忠実度の高いomni-79ヌクレアーゼバリアント
JP2024540558A (ja) 新規なomni crisprヌクレアーゼ
JP2024506608A (ja) Omni-103 crisprヌクレアーゼ
JP2023546694A (ja) 新規のomni56、58、65、68、71、75、78及び84crisprヌクレアーゼ
JP2026501585A (ja) Omni-335 crisprヌクレアーゼ
JP2026501682A (ja) Omni xl1~22 crisprヌクレアーゼ
HK40115053A (zh) 新型omni crispr核酸酶
HK40111655A (zh) 新型omni 115、124、127、144-149、159、218、237、248、251-253和259 crispr核酸酶
HK40116323A (zh) Omni-103 crispr核酸酶-rna复合物
HK40109205A (zh) Omni90-99、101、104-110、114、116、118-123、125、126、128、129和131-138crispr核酸酶
CN117597142A (zh) Omni 90-99、101、104-110、114、116、118-123、125、126、128、129和131-138 crispr核酸酶
HK40115104A (zh) 工程化高活性omni-79核酸酶变体

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241217

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251205

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20251205