CN117980315A - 1’-烷基修饰的核糖衍生物及使用方法 - Google Patents

1’-烷基修饰的核糖衍生物及使用方法 Download PDF

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Publication number
CN117980315A
CN117980315A CN202280061450.4A CN202280061450A CN117980315A CN 117980315 A CN117980315 A CN 117980315A CN 202280061450 A CN202280061450 A CN 202280061450A CN 117980315 A CN117980315 A CN 117980315A
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compound
scaffold
conjugate
alkyl
formula
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CN202280061450.4A
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Chinese (zh)
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王为民
蔡晓川
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Saint Germain Biotech Inc
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Saint Germain Biotech Inc
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/26Acyclic or carbocyclic radicals, substituted by hetero rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D307/00Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom
    • C07D307/02Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings
    • C07D307/04Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
    • C07D307/18Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D307/20Oxygen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/655Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having oxygen atoms, with or without sulfur, selenium, or tellurium atoms, as the only ring hetero atoms
    • C07F9/65515Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having oxygen atoms, with or without sulfur, selenium, or tellurium atoms, as the only ring hetero atoms the oxygen atom being part of a five-membered ring
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
    • C07F9/65586Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system at least one of the hetero rings does not contain nitrogen as ring hetero atom
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H13/00Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids
    • 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
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202280061450.4A 2021-08-05 2022-08-05 1’-烷基修饰的核糖衍生物及使用方法 Pending CN117980315A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163229628P 2021-08-05 2021-08-05
US63/229,628 2021-08-05
PCT/US2022/039517 WO2023014938A1 (en) 2021-08-05 2022-08-05 1'-alkyl modified ribose derivatives and methods of use

Publications (1)

Publication Number Publication Date
CN117980315A true CN117980315A (zh) 2024-05-03

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Family Applications (1)

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CN202280061450.4A Pending CN117980315A (zh) 2021-08-05 2022-08-05 1’-烷基修饰的核糖衍生物及使用方法

Country Status (10)

Country Link
US (2) US12465647B2 (https=)
EP (1) EP4380946A1 (https=)
JP (1) JP7842850B2 (https=)
KR (1) KR20240070519A (https=)
CN (1) CN117980315A (https=)
AU (1) AU2022324471A1 (https=)
CA (1) CA3227661A1 (https=)
IL (1) IL310547A (https=)
MX (1) MX2024001581A (https=)
WO (1) WO2023014938A1 (https=)

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WO2025256544A1 (zh) * 2024-06-11 2025-12-18 福建盛迪医药有限公司 一种1-氨烷基修饰的呋喃核糖衍生物的制备方法

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MX2025013094A (es) * 2023-05-03 2026-02-03 Sanegene Bio Usa Inc Conjugados basados en lipidos para la administracion sistemica en el sistema nervioso central, el sistema nervioso periferico y ocular
US20250064939A1 (en) * 2023-07-24 2025-02-27 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
CN116854754B (zh) 2023-09-01 2023-12-12 北京悦康科创医药科技股份有限公司 一种含有核糖环或其衍生结构的GalNAc化合物及其寡核苷酸缀合物
CN121263426A (zh) * 2024-05-09 2026-01-02 石药集团中奇制药技术(石家庄)有限公司 GalNAc衍生物及其寡聚核苷酸缀合物
US20250382616A1 (en) * 2024-06-18 2025-12-18 Sanegene Bio Usa Inc. Small interfering rna targeting cfb and uses thereof
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WO2026030554A1 (en) * 2024-07-31 2026-02-05 Sanegene Bio Usa Inc. Saccharide-based conjugates for rna delivery and therapy
CN118638166B (zh) 2024-08-15 2024-12-17 北京悦康科创医药科技股份有限公司 含有三氮唑结构的GalNAc化合物及其寡核苷酸缀合物
WO2026041067A2 (en) 2024-08-20 2026-02-26 Sanegene Bio Usa Inc. Small interfering rna targeting myostatin (mstn) and uses thereof
WO2026046411A1 (en) 2024-09-02 2026-03-05 Sanegene Bio Usa Inc. Small interfering rna targeting plasminogen (plg) and uses thereof

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025256544A1 (zh) * 2024-06-11 2025-12-18 福建盛迪医药有限公司 一种1-氨烷基修饰的呋喃核糖衍生物的制备方法

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JP7842850B2 (ja) 2026-04-08
US20260021188A1 (en) 2026-01-22
IL310547A (en) 2024-03-01
US20230138928A1 (en) 2023-05-04
US12465647B2 (en) 2025-11-11
WO2023014938A1 (en) 2023-02-09
EP4380946A1 (en) 2024-06-12
MX2024001581A (es) 2024-04-18
CA3227661A1 (en) 2023-02-09
KR20240070519A (ko) 2024-05-21
JP2024529067A (ja) 2024-08-01
AU2022324471A1 (en) 2024-02-15

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