CA3119580A1 - Crosslinked artificial nucleic acid alna - Google Patents

Crosslinked artificial nucleic acid alna Download PDF

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Publication number
CA3119580A1
CA3119580A1 CA3119580A CA3119580A CA3119580A1 CA 3119580 A1 CA3119580 A1 CA 3119580A1 CA 3119580 A CA3119580 A CA 3119580A CA 3119580 A CA3119580 A CA 3119580A CA 3119580 A1 CA3119580 A1 CA 3119580A1
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CA
Canada
Prior art keywords
group
substituents
optionally substituted
methyl
oxa
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.)
Pending
Application number
CA3119580A
Other languages
English (en)
French (fr)
Inventor
Hiroaki Sawamoto
Shinji Kumagai
Hiroyuki Furukawa
Tomo Araki
Masayuki Utsugi
Satoshi Obika
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.)
Tanabe Pharma Corp
Original Assignee
Osaka University NUC
Mitsubishi Tanabe Pharma Corp
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 Osaka University NUC, Mitsubishi Tanabe Pharma Corp filed Critical Osaka University NUC
Publication of CA3119580A1 publication Critical patent/CA3119580A1/en
Pending legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06Pyrimidine radicals
    • C07H19/10Pyrimidine radicals with the saccharide radical esterified by phosphoric or polyphosphoric 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
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06Pyrimidine radicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16Purine radicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16Purine radicals
    • C07H19/20Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds 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
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02PCLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00Technologies relating to chemical industry
    • Y02P20/50Improvements relating to the production of bulk chemicals
    • Y02P20/55Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3119580A 2018-11-12 2019-11-11 Crosslinked artificial nucleic acid alna Pending CA3119580A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2018212424 2018-11-12
JP2018-212424 2018-11-12
PCT/JP2019/044182 WO2020100826A1 (ja) 2018-11-12 2019-11-11 架橋型人工核酸alna

Publications (1)

Publication Number Publication Date
CA3119580A1 true CA3119580A1 (en) 2020-05-22

Family

ID=70731902

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3119580A Pending CA3119580A1 (en) 2018-11-12 2019-11-11 Crosslinked artificial nucleic acid alna

Country Status (14)

Country Link
US (2) US12338265B2 (https=)
EP (3) EP4275690A3 (https=)
JP (1) JP7356448B2 (https=)
KR (1) KR102886120B1 (https=)
CN (4) CN119119156A (https=)
AU (1) AU2019381386C1 (https=)
BR (1) BR112021009168A2 (https=)
CA (1) CA3119580A1 (https=)
IL (2) IL283103B2 (https=)
MX (1) MX2021005537A (https=)
MY (1) MY208522A (https=)
PH (1) PH12021551082A1 (https=)
SG (1) SG11202104901VA (https=)
WO (1) WO2020100826A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7776420B2 (ja) * 2020-05-12 2025-11-26 田辺三菱製薬株式会社 Ataxin 3発現を調節するための化合物、方法及び医薬組成物
JP2023130528A (ja) * 2020-06-24 2023-09-21 田辺三菱製薬株式会社 Plp1発現を調節するための化合物、方法及び医薬組成物
JPWO2022014703A1 (https=) 2020-07-17 2022-01-20
WO2024101446A1 (ja) 2022-11-10 2024-05-16 ルクサナバイオテク株式会社 架橋部にグアニジノ構造を有する修飾ヌクレオシドおよびそれを用いたオリゴヌクレオチドの製造方法
CN120418261A (zh) 2022-12-21 2025-08-01 田边三菱制药株式会社 制造中间体的制造方法、该制造中间体及使用其的桥连型人工核酸中间体的制造方法
WO2025206092A1 (ja) * 2024-03-27 2025-10-02 田辺三菱製薬株式会社 Myoregulin発現を調節するための化合物及び医薬組成物

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4236812B2 (ja) * 1997-09-12 2009-03-11 エクシコン エ/エス オリゴヌクレオチド類似体
AU9393798A (en) 1997-09-17 1999-04-05 Affymetrix, Inc. Genetic compositions and methods
WO2007145593A1 (en) * 2006-06-15 2007-12-21 Jyoti Chattopadhyaya Conformationally constrained 2' -n,4' -c-ethylene-bridged thymidine (aza-ena-t)
EP2580228B1 (en) * 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
BR112013026345A2 (pt) 2011-04-13 2019-04-24 Merck Sharp & Dohe Corp. composto, composição farmacêutica, uso de um composto, e, método para tratar um paciente infectado com hcv
BR112014001075A2 (pt) 2011-07-19 2016-11-29 Univ Idaho sonda, seu uso, método in vitro para associar uma sonda com um alvo e kit
KR101707437B1 (ko) * 2012-09-21 2017-02-16 고꾸리쯔 다이가꾸 호우징 오사까 다이가꾸 구아니딘 가교를 갖는 인공 뉴클레오시드 및 올리고뉴클레오티드
WO2014124952A1 (en) 2013-02-12 2014-08-21 Syddansk Universitet "clickable" alkyne-lna oligonucleotides
WO2016128583A2 (en) 2015-02-15 2016-08-18 Ribo Task Aps Acyl-amino-lna and/or hydrocarbyl-amino-lna oligonucleotides
TW201718618A (zh) * 2015-09-18 2017-06-01 田邊三菱製藥股份有限公司 架橋型核酸GuNA,其製造方法,及中間體化合物
WO2020203880A1 (ja) 2019-03-29 2020-10-08 田辺三菱製薬株式会社 Dux4の発現を調節するための化合物、方法及び医薬組成物

Also Published As

Publication number Publication date
PH12021551082A1 (en) 2021-11-22
IL283103A (en) 2021-06-30
WO2020100826A1 (ja) 2020-05-22
JPWO2020100826A1 (ja) 2021-09-30
KR102886120B1 (ko) 2025-11-14
KR20210090659A (ko) 2021-07-20
EP3881851A4 (en) 2023-02-15
BR112021009168A2 (pt) 2021-08-17
CN112996522A (zh) 2021-06-18
MY208522A (en) 2025-05-14
US20250340584A1 (en) 2025-11-06
CN112996522B (zh) 2024-09-20
IL316158B1 (en) 2026-04-01
CN117964673A (zh) 2024-05-03
MX2021005537A (es) 2021-09-10
CN117964674A (zh) 2024-05-03
EP4275690A2 (en) 2023-11-15
AU2019381386A1 (en) 2021-06-17
EP4275690A3 (en) 2023-11-22
SG11202104901VA (en) 2021-06-29
AU2019381386C1 (en) 2025-11-27
AU2019381386B2 (en) 2025-06-26
IL283103B2 (en) 2025-04-01
US12338265B2 (en) 2025-06-24
EP4275689A2 (en) 2023-11-15
EP4275689A3 (en) 2023-11-22
JP7356448B2 (ja) 2023-10-04
IL316158A (en) 2024-12-01
US20220002336A1 (en) 2022-01-06
IL283103B1 (en) 2024-12-01
CN119119156A (zh) 2024-12-13
EP3881851A1 (en) 2021-09-22

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