JPWO2024225425A1 - - Google Patents

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Publication number
JPWO2024225425A1
JPWO2024225425A1 JP2024551649A JP2024551649A JPWO2024225425A1 JP WO2024225425 A1 JPWO2024225425 A1 JP WO2024225425A1 JP 2024551649 A JP2024551649 A JP 2024551649A JP 2024551649 A JP2024551649 A JP 2024551649A JP WO2024225425 A1 JPWO2024225425 A1 JP WO2024225425A1
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JP
Japan
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JP2024551649A
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Japanese (ja)
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JPWO2024225425A5 (https=
JP7660960B2 (ja
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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • C12N15/87—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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2024551649A 2023-04-26 2024-04-26 標的ヌクレオチド配列の改変のための非天然型ポリヌクレオチド Active JP7660960B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025051903A JP2025092562A (ja) 2023-04-26 2025-03-26 標的ヌクレオチド配列の改変のための非天然型ポリヌクレオチド

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2023071927 2023-04-26
JP2023071927 2023-04-26
PCT/JP2024/016395 WO2024225425A1 (ja) 2023-04-26 2024-04-26 標的ヌクレオチド配列の改変のための非天然型ポリヌクレオチド

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025051903A Division JP2025092562A (ja) 2023-04-26 2025-03-26 標的ヌクレオチド配列の改変のための非天然型ポリヌクレオチド

Publications (3)

Publication Number Publication Date
JPWO2024225425A1 true JPWO2024225425A1 (https=) 2024-10-31
JPWO2024225425A5 JPWO2024225425A5 (https=) 2025-04-02
JP7660960B2 JP7660960B2 (ja) 2025-04-14

Family

ID=93256810

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2024551649A Active JP7660960B2 (ja) 2023-04-26 2024-04-26 標的ヌクレオチド配列の改変のための非天然型ポリヌクレオチド
JP2025051903A Pending JP2025092562A (ja) 2023-04-26 2025-03-26 標的ヌクレオチド配列の改変のための非天然型ポリヌクレオチド

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025051903A Pending JP2025092562A (ja) 2023-04-26 2025-03-26 標的ヌクレオチド配列の改変のための非天然型ポリヌクレオチド

Country Status (7)

Country Link
EP (1) EP4703472A1 (https=)
JP (2) JP7660960B2 (https=)
KR (1) KR20250168544A (https=)
CN (1) CN121443735A (https=)
AU (1) AU2024260090A1 (https=)
IL (1) IL324176A (https=)
WO (1) WO2024225425A1 (https=)

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009520495A (ja) * 2005-12-22 2009-05-28 キージーン ナムローゼ フェンノートシャップ 改善された標的化ヌクレオチド交換のための代替ヌクレオチド
JP2010530750A (ja) * 2007-06-22 2010-09-16 キージーン・エン・フェー 改善された修飾オリゴヌクレオチドを用いた標的ヌクレオチドの交換
JP2011062207A (ja) * 2004-07-07 2011-03-31 National Institute Of Agrobiological Sciences 核酸を配列特異的に標識する方法、およびそれを利用した新規核酸検出法
JP2016517273A (ja) * 2013-03-14 2016-06-16 ソマロジック, インコーポレイテッドSomaLogic, Inc. Ｉｌ−６に結合するアプタマー及びｉｌ−６介在性状態の治療または診断におけるそれらの使用
JP2020500035A (ja) * 2016-11-11 2020-01-09 バイオ−ラド ラボラトリーズ，インコーポレイティド 核酸試料をプロセシングする方法
WO2022256440A2 (en) * 2021-06-01 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102016035A (zh) * 2006-11-02 2011-04-13 犹他大学研究基金会 可用于等位基因特异pcr的寡聚核苷酸
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
EP3115457B1 (en) 2014-03-05 2019-10-02 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
DK3636753T5 (da) 2017-06-08 2024-08-26 Univ Osaka Fremgangsmåde til fremstilling af DNA-redigeret eukaryotisk celle

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011062207A (ja) * 2004-07-07 2011-03-31 National Institute Of Agrobiological Sciences 核酸を配列特異的に標識する方法、およびそれを利用した新規核酸検出法
JP2009520495A (ja) * 2005-12-22 2009-05-28 キージーン ナムローゼ フェンノートシャップ 改善された標的化ヌクレオチド交換のための代替ヌクレオチド
JP2010530750A (ja) * 2007-06-22 2010-09-16 キージーン・エン・フェー 改善された修飾オリゴヌクレオチドを用いた標的ヌクレオチドの交換
JP2016517273A (ja) * 2013-03-14 2016-06-16 ソマロジック, インコーポレイテッドSomaLogic, Inc. Ｉｌ−６に結合するアプタマー及びｉｌ−６介在性状態の治療または診断におけるそれらの使用
JP2020500035A (ja) * 2016-11-11 2020-01-09 バイオ−ラド ラボラトリーズ，インコーポレイティド 核酸試料をプロセシングする方法
WO2022256440A2 (en) * 2021-06-01 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof

Also Published As

Publication number Publication date
AU2024260090A1 (en) 2025-12-04
IL324176A (en) 2025-12-01
EP4703472A1 (en) 2026-03-04
WO2024225425A1 (ja) 2024-10-31
KR20250168544A (ko) 2025-12-02
JP7660960B2 (ja) 2025-04-14
JP2025092562A (ja) 2025-06-19
CN121443735A (zh) 2026-01-30

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