JP7793144B2 - 修飾ヘテロ核酸 - Google Patents

修飾ヘテロ核酸

Info

Publication number
JP7793144B2
JP7793144B2 JP2021551735A JP2021551735A JP7793144B2 JP 7793144 B2 JP7793144 B2 JP 7793144B2 JP 2021551735 A JP2021551735 A JP 2021551735A JP 2021551735 A JP2021551735 A JP 2021551735A JP 7793144 B2 JP7793144 B2 JP 7793144B2
Authority
JP
Japan
Prior art keywords
nucleic acid
modified
acid strand
sugar
region
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
JP2021551735A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2021070959A1 (https=
Inventor
隆徳 横田
哲也 永田
大貴 山田
英紀 古川
能紀 余郷
健一 宮田
章夫 内田
直輝 冨田
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.)
Takeda Pharmaceutical Co Ltd
Tokyo Institute of Technology NUC
Institute of Science Tokyo
Original Assignee
Takeda Pharmaceutical Co Ltd
Tokyo Institute of Technology NUC
Institute of Science Tokyo
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 Takeda Pharmaceutical Co Ltd, Tokyo Institute of Technology NUC, Institute of Science Tokyo filed Critical Takeda Pharmaceutical Co Ltd
Publication of JPWO2021070959A1 publication Critical patent/JPWO2021070959A1/ja
Priority to JP2025130417A priority Critical patent/JP2025156522A/ja
Application granted granted Critical
Publication of JP7793144B2 publication Critical patent/JP7793144B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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
    • A61K31/713—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

Landscapes

  • 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)
JP2021551735A 2019-10-11 2020-10-09 修飾ヘテロ核酸 Active JP7793144B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025130417A JP2025156522A (ja) 2019-10-11 2025-08-05 修飾ヘテロ核酸

Applications Claiming Priority (3)

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025130417A Division JP2025156522A (ja) 2019-10-11 2025-08-05 修飾ヘテロ核酸

Publications (2)

Publication Number Publication Date
JPWO2021070959A1 JPWO2021070959A1 (https=) 2021-04-15
JP7793144B2 true JP7793144B2 (ja) 2026-01-05

Family

ID=75438209

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021551735A Active JP7793144B2 (ja) 2019-10-11 2020-10-09 修飾ヘテロ核酸
JP2025130417A Pending JP2025156522A (ja) 2019-10-11 2025-08-05 修飾ヘテロ核酸

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025130417A Pending JP2025156522A (ja) 2019-10-11 2025-08-05 修飾ヘテロ核酸

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 (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015502134A (ja) 2011-12-16 2015-01-22 国立大学法人 東京医科歯科大学 キメラ２重鎖核酸
WO2018056442A1 (ja) 2016-09-23 2018-03-29 国立大学法人東京医科歯科大学 血液脳関門通過型ヘテロ２本鎖核酸
WO2018062510A1 (ja) 2016-09-29 2018-04-05 国立大学法人東京医科歯科大学 オーバーハングを有する二本鎖核酸複合体
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
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2019118916A1 (en) 2017-12-14 2019-06-20 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use

Family Cites Families (12)

* Cited by examiner, † Cited by third party
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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
EP3004347B1 (en) * 2013-05-30 2018-09-26 National University Corporation Tokyo Medical and Dental University Double-stranded agents for delivering therapeutic oligonucleotides
AU2014282666A1 (en) * 2013-06-16 2016-01-07 National University Corporation Tokyo Medical And Dental University Double-stranded antisense nucleic acid with exon-skipping effect
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
EP3647423A4 (en) * 2017-06-30 2021-03-24 National University Corporation Tokyo Medical and Dental University DOUBLE STRANDED HETERO-ANTI-MIRNA
JP6574868B1 (ja) 2018-04-27 2019-09-11 株式会社大一商会 遊技機

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015502134A (ja) 2011-12-16 2015-01-22 国立大学法人 東京医科歯科大学 キメラ２重鎖核酸
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
WO2018056442A1 (ja) 2016-09-23 2018-03-29 国立大学法人東京医科歯科大学 血液脳関門通過型ヘテロ２本鎖核酸
WO2018062510A1 (ja) 2016-09-29 2018-04-05 国立大学法人東京医科歯科大学 オーバーハングを有する二本鎖核酸複合体
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2019118916A1 (en) 2017-12-14 2019-06-20 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use

Also Published As

Publication number Publication date
EP4043565A1 (en) 2022-08-17
CN115103911A (zh) 2022-09-23
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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