KR20240026181A - 폴리뉴클레오티드 및 의약 조성물 - Google Patents

폴리뉴클레오티드 및 의약 조성물 Download PDF

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Publication number
KR20240026181A
KR20240026181A KR1020247001879A KR20247001879A KR20240026181A KR 20240026181 A KR20240026181 A KR 20240026181A KR 1020247001879 A KR1020247001879 A KR 1020247001879A KR 20247001879 A KR20247001879 A KR 20247001879A KR 20240026181 A KR20240026181 A KR 20240026181A
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South Korea
Prior art keywords
modified
nucleotides
sugar
nucleotide
polynucleotide
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Pending
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KR1020247001879A
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English (en)
Korean (ko)
Inventor
히로토 이와이
마사카즈 홈마
다카유키 아타고
준이치로 야마모토
히로시 아베
야스아키 기무라
Original Assignee
쿄와 기린 가부시키가이샤
내셔널 유니버시티 코포레이션 토카이 내셔널 하이어 에듀케이션 앤드 리서치 시스템
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Application filed by 쿄와 기린 가부시키가이샤, 내셔널 유니버시티 코포레이션 토카이 내셔널 하이어 에듀케이션 앤드 리서치 시스템 filed Critical 쿄와 기린 가부시키가이샤
Publication of KR20240026181A publication Critical patent/KR20240026181A/ko
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • 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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
KR1020247001879A 2021-06-30 2022-06-30 폴리뉴클레오티드 및 의약 조성물 Pending KR20240026181A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
JP2021109239 2021-06-30
JPJP-P-2021-109239 2021-06-30
JPJP-P-2021-169846 2021-10-15
JP2021169846 2021-10-15
PCT/JP2022/026411 WO2023277168A1 (ja) 2021-06-30 2022-06-30 ポリヌクレオチド及び医薬組成物

Publications (1)

Publication Number Publication Date
KR20240026181A true KR20240026181A (ko) 2024-02-27

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247001879A Pending KR20240026181A (ko) 2021-06-30 2022-06-30 폴리뉴클레오티드 및 의약 조성물

Country Status (7)

Country Link
US (1) US20240325426A1 (https=)
EP (1) EP4365288A4 (https=)
JP (1) JPWO2023277168A1 (https=)
KR (1) KR20240026181A (https=)
AU (1) AU2022302611A1 (https=)
CA (1) CA3225964A1 (https=)
WO (1) WO2023277168A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPWO2024185697A1 (https=) * 2023-03-03 2024-09-12
WO2025111430A1 (en) * 2023-11-21 2025-05-30 Prime Medicine, Inc. CHEMICAL MODIFICATIONS IN mRNA POLY(A) TAIL
WO2026010855A1 (en) * 2024-07-02 2026-01-08 Trilink Biotechnologies, Llc Modified rna for increasing protein expression

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH01114346A (ja) 1987-10-26 1989-05-08 Seiko Electronic Components Ltd 冷却ファン付モータ
JP2014093574A (ja) 2012-10-31 2014-05-19 Kyocera Document Solutions Inc 画像形成装置及び画像形成プログラム
JP2016022914A (ja) 2014-07-24 2016-02-08 ヤンマー株式会社 作業車輌

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US20030083272A1 (en) * 1997-09-19 2003-05-01 Lahive & Cockfield, Llp Sense mrna therapy
EP2283132B1 (en) * 2008-05-02 2016-10-26 Epicentre Technologies Corporation Selective 5' ligation tagging of rna
EP2694660B1 (en) * 2011-04-03 2018-08-08 The General Hospital Corporation Efficient protein expression in vivo using modified rna (mod-rna)
US20140200261A1 (en) * 2013-01-17 2014-07-17 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
JP2017523777A (ja) * 2014-07-17 2017-08-24 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． ポリヌクレオチドの末端修飾
WO2016022914A1 (en) 2014-08-08 2016-02-11 Moderna Therapeutics, Inc. Compositions and methods for the treatment of ophthalmic diseases and conditions
EP3233132A4 (en) * 2014-12-19 2018-06-27 Modernatx, Inc. Terminal modifications of polynucleotides
RS67188B1 (sr) 2016-01-11 2025-10-31 Innate Tumor Immunity Inc Ciklični dinukleotidi za lečenje stanja povezanih sa aktivnošću sting kao što je kancer
JP7787514B2 (ja) * 2019-12-26 2025-12-17 国立大学法人東海国立大学機構 ポリヌクレオチド及び医薬組成物

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH01114346A (ja) 1987-10-26 1989-05-08 Seiko Electronic Components Ltd 冷却ファン付モータ
JP2014093574A (ja) 2012-10-31 2014-05-19 Kyocera Document Solutions Inc 画像形成装置及び画像形成プログラム
JP2016022914A (ja) 2014-07-24 2016-02-08 ヤンマー株式会社 作業車輌

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
비특허문헌 1: 네이쳐 리뷰즈 드러그 디스커버리(Nature Reviews Drug Discovery), 제13권, 759-780 페이지 (2014)
비특허문헌 2: 네이쳐 바이오테크놀로지(Nature Biotechnology), 제35권, 제3호, 238-248 페이지 (2017)
비특허문헌 3: 드러그 디스커버리 투데이(Drug Discovery Today), 제13권, 제19/20호, 842-855 페이지 (2008)
비특허문헌 4: 네이쳐 바이오테크놀로지(Nature Biotechnology), 제35권, 제3호, 193-197 페이지 (2017)
비특허문헌 5: 네이쳐(Nature), 제547권, 제7662호, 222-226 페이지 (2017)
비특허문헌 6: 뉴클레익 애시즈 리서치(Nucleic Acids Research), 제44권, 제2호, 852-862 페이지 (2015)
비특허문헌 7: 진즈(Genes), 제10권, 제2호, 84 페이지 (2019)

Also Published As

Publication number Publication date
EP4365288A4 (en) 2025-03-12
WO2023277168A1 (ja) 2023-01-05
CA3225964A1 (en) 2023-01-25
US20240325426A1 (en) 2024-10-03
JPWO2023277168A1 (https=) 2023-01-05
EP4365288A1 (en) 2024-05-08
AU2022302611A1 (en) 2024-02-15

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