JP7660960B2 - 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 - Google Patents

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 Download PDF

Info

Publication number
JP7660960B2
JP7660960B2 JP2024551649A JP2024551649A JP7660960B2 JP 7660960 B2 JP7660960 B2 JP 7660960B2 JP 2024551649 A JP2024551649 A JP 2024551649A JP 2024551649 A JP2024551649 A JP 2024551649A JP 7660960 B2 JP7660960 B2 JP 7660960B2
Authority
JP
Japan
Prior art keywords
nucleotide
nucleotides
geo
lna
natural
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.)
Active
Application number
JP2024551649A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2024225425A5 (https=
JPWO2024225425A1 (https=
Inventor
貴人 能見
圭子 早見
直紀 井上
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.)
Eurus Therapeutics
Original Assignee
Eurus Therapeutics
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 Eurus Therapeutics filed Critical Eurus Therapeutics
Publication of JPWO2024225425A1 publication Critical patent/JPWO2024225425A1/ja
Priority to JP2025051903A priority Critical patent/JP2025092562A/ja
Publication of JPWO2024225425A5 publication Critical patent/JPWO2024225425A5/ja
Application granted granted Critical
Publication of JP7660960B2 publication Critical patent/JP7660960B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • C12N15/87Introduction 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
    • 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
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
    • 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

  • 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 JPWO2024225425A1 (https=) 2024-10-31
JPWO2024225425A5 JPWO2024225425A5 (https=) 2025-04-02
JP7660960B2 true 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 (5)

* 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

Family Cites Families (5)

* 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
EP4347818A2 (en) * 2021-06-01 2024-04-10 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof

Patent Citations (5)

* 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

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
JPWO2024225425A1 (https=) 2024-10-31
JP2025092562A (ja) 2025-06-19
CN121443735A (zh) 2026-01-30

Similar Documents

Publication Publication Date Title
US11560555B2 (en) Engineered proteins
AU2017320901C1 (en) Chemically modified single-stranded RNA-editing oligonucleotides
KR102691636B1 (ko) 상동 재조합에 의한 crispr/cas-기반 게놈 편집을 위한 화합물 및 방법
CA2968336C (en) Construct for site directed editing of an adenosine nucleotide in target rna
CA3136735A1 (en) Methods and compositions for editing rnas
GB2617658A (en) Class II, type V CRISPR systems
KR20240031238A (ko) Crispr 뉴클레이스를 포함하는 유전자 편집 시스템 및 이의 용도
WO2018117746A1 (ko) 동물 배아의 염기 교정용 조성물 및 염기 교정 방법
US20240218339A1 (en) Class ii, type v crispr systems
WO2023086834A1 (en) Direct replacement genome editing
KR102679001B1 (ko) 신규의 개량된 염기 편집 또는 교정용 융합단백질 및 이의 용도
JP7660960B2 (ja) 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
EP4504221A2 (en) Compositions and methods for generating cells with reduced immunogenicty
CA3245468A1 (en) Compositions, systems and methods for eukaryotic gene editing
WO2026094932A1 (ja) 標的ヌクレオチド配列の改変のための二本鎖ヌクレオチド複合体
US20250290056A1 (en) Modrna-based cas endonuclease and base editor and uses thereof
US20240425850A1 (en) Noncanonical crRNA for Highly Efficient Genome Editing
EP4601650A2 (en) Compositions and methods for targeting, editing, or modifying genes
WO2026050560A1 (en) Cas9 fusion proteins with internal nls: compositions and methods
WO2024220715A2 (en) Effector proteins and uses thereof
WO2025101679A1 (en) Effector proteins and uses thereof
WO2024107836A1 (en) Methods for intron-driven gene editing and control

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240829

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240829

A871 Explanation of circumstances concerning accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A871

Effective date: 20240829

AA64 Notification of invalidation of claim of internal priority (with term)

Free format text: JAPANESE INTERMEDIATE CODE: A241764

Effective date: 20240909

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240911

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241218

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250127

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250228

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250326

R150 Certificate of patent or registration of utility model

Ref document number: 7660960

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150