CN115103911A - 修饰的异源核酸 - Google Patents

修饰的异源核酸 Download PDF

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Publication number
CN115103911A
CN115103911A CN202080084535.5A CN202080084535A CN115103911A CN 115103911 A CN115103911 A CN 115103911A CN 202080084535 A CN202080084535 A CN 202080084535A CN 115103911 A CN115103911 A CN 115103911A
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China
Prior art keywords
nucleic acid
modified
sugar
region
acid strand
Prior art date
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Pending
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CN202080084535.5A
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English (en)
Chinese (zh)
Inventor
横田隆徳
永田哲也
山田大贵
古川英纪
余郷能纪
宫田健一
内田章夫
冨田直辉
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Takeda Pharmaceutical Co Ltd
Tokyo Medical and Dental University NUC
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Takeda Pharmaceutical Co Ltd
Tokyo Medical and Dental University NUC
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Publication date
Application filed by Takeda Pharmaceutical Co Ltd, Tokyo Medical and Dental University NUC filed Critical Takeda Pharmaceutical Co Ltd
Publication of CN115103911A publication Critical patent/CN115103911A/zh
Pending legal-status Critical Current

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    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • 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
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
CN202080084535.5A 2019-10-11 2020-10-09 修饰的异源核酸 Pending CN115103911A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019188042 2019-10-11
JP2019-188042 2019-10-11
PCT/JP2020/038380 WO2021070959A1 (ja) 2019-10-11 2020-10-09 修飾ヘテロ核酸

Publications (1)

Publication Number Publication Date
CN115103911A true CN115103911A (zh) 2022-09-23

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ID=75438209

Family Applications (1)

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CN202080084535.5A Pending CN115103911A (zh) 2019-10-11 2020-10-09 修饰的异源核酸

Country Status (5)

Country Link
US (1) US20240117346A1 (https=)
EP (1) EP4043565A4 (https=)
JP (2) JP7793144B2 (https=)
CN (1) CN115103911A (https=)
WO (1) WO2021070959A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4349986A4 (en) * 2021-05-25 2025-11-19 Univ Nat Corp Tokyo Medical & Dental HETERONUCLEIC ACID WITH A CONTENT OF ?????? OR ????
JPWO2023022229A1 (https=) * 2021-08-19 2023-02-23
JP2024089860A (ja) * 2022-12-22 2024-07-04 学校法人東京薬科大学 Ｚｉｃ５発現を調節するための核酸複合体

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104126010A (zh) * 2011-12-16 2014-10-29 国立大学法人东京医科齿科大学 嵌合的双链核酸
CN105264073A (zh) * 2013-05-30 2016-01-20 国立大学法人东京医科齿科大学 用于递送治疗性寡核苷酸的双链剂
CN105324119A (zh) * 2013-06-16 2016-02-10 国立大学法人东京医科齿科大学 具有外显子跳跃效应的双链反义核酸
WO2018223056A1 (en) * 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2019004420A1 (ja) * 2017-06-30 2019-01-03 国立大学法人東京医科歯科大学 ヘテロ二本鎖型ａｎｔｉｍｉＲ
WO2019118916A1 (en) * 2017-12-14 2019-06-20 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US20190240352A1 (en) * 2016-09-29 2019-08-08 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
US20190247414A1 (en) * 2016-09-23 2019-08-15 National University Corporation Tokyo Medical And Dental University Blood-brain barrier permeable heteroduplex nucleic acid

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5955599A (en) 1995-06-01 1999-09-21 Hybridon, Inc. Process for making oligonucleotides containing o- and s- methylphosphotriester internucleoside linkages
JP2009536222A (ja) 2006-05-05 2009-10-08 アイシス ファーマシューティカルズ， インコーポレーテッド Ｐｃｓｋ９の発現を調節するための化合物および方法
CA2666191C (en) 2006-10-09 2017-07-11 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9
ES2526295T5 (es) 2006-10-18 2021-05-04 Ionis Pharmaceuticals Inc Compuestos antisentido
WO2015168172A1 (en) 2014-04-28 2015-11-05 Isis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
WO2016081600A1 (en) 2014-11-18 2016-05-26 Zata Pharmaceuticals, Inc. Phosphoramidite synthones for the synthesis of self-neutralizing oligonucleotide compounds
JP2018528783A (ja) * 2015-09-25 2018-10-04 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． コンジュゲートアンチセンス化合物及びその使用
JP6574868B1 (ja) 2018-04-27 2019-09-11 株式会社大一商会 遊技機

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104126010A (zh) * 2011-12-16 2014-10-29 国立大学法人东京医科齿科大学 嵌合的双链核酸
CN105264073A (zh) * 2013-05-30 2016-01-20 国立大学法人东京医科齿科大学 用于递送治疗性寡核苷酸的双链剂
CN105324119A (zh) * 2013-06-16 2016-02-10 国立大学法人东京医科齿科大学 具有外显子跳跃效应的双链反义核酸
US20190247414A1 (en) * 2016-09-23 2019-08-15 National University Corporation Tokyo Medical And Dental University Blood-brain barrier permeable heteroduplex nucleic acid
US20190240352A1 (en) * 2016-09-29 2019-08-08 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
WO2018223056A1 (en) * 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2019004420A1 (ja) * 2017-06-30 2019-01-03 国立大学法人東京医科歯科大学 ヘテロ二本鎖型ａｎｔｉｍｉＲ
WO2019118916A1 (en) * 2017-12-14 2019-06-20 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use

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Publication number Publication date
JP7793144B2 (ja) 2026-01-05
EP4043565A1 (en) 2022-08-17
JPWO2021070959A1 (https=) 2021-04-15
EP4043565A4 (en) 2024-01-24
JP2025156522A (ja) 2025-10-14
WO2021070959A1 (ja) 2021-04-15
US20240117346A1 (en) 2024-04-11

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