JP7842850B2 - 1’-アルキル修飾リボース誘導体及び使用方法 - Google Patents

1’-アルキル修飾リボース誘導体及び使用方法

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Publication number
JP7842850B2
JP7842850B2 JP2024507164A JP2024507164A JP7842850B2 JP 7842850 B2 JP7842850 B2 JP 7842850B2 JP 2024507164 A JP2024507164 A JP 2024507164A JP 2024507164 A JP2024507164 A JP 2024507164A JP 7842850 B2 JP7842850 B2 JP 7842850B2
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alkyl
optionally substituted
conjugate
halogens
compound
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Japanese (ja)
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JP2024529067A (ja
JP2024529067A5 (enExample
Inventor
ワン ウェイミン
ツァイ シアオチョアン
Original Assignee
セーンジーン バイオ ユーエスエー インコーポレイティド
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Publication of JP2024529067A publication Critical patent/JP2024529067A/ja
Publication of JP2024529067A5 publication Critical patent/JP2024529067A5/ja
Priority to JP2026054255A priority Critical patent/JP2026095525A/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D307/00—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom
    • C07D307/02—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings
    • C07D307/04—Heterocyclic 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
    • C07D307/18—Heterocyclic 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
    • C07D307/20—Oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/655—Heterocyclic 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
    • C07F9/65515—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558—Heterocyclic 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
    • C07F9/65586—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H13/00—Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/26—Acyclic or carbocyclic radicals, substituted by hetero rings
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • 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)
JP2024507164A 2021-08-05 2022-08-05 1’-アルキル修飾リボース誘導体及び使用方法 Active JP7842850B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2026054255A JP2026095525A (ja) 2021-08-05 2026-03-27 1’-アルキル修飾リボース誘導体及び使用方法

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

Related Child Applications (1)

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Publications (3)

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JP2024529067A JP2024529067A (ja) 2024-08-01
JP2024529067A5 JP2024529067A5 (enExample) 2025-08-08
JP7842850B2 true JP7842850B2 (ja) 2026-04-08

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JP2026054255A Pending JP2026095525A (ja) 2021-08-05 2026-03-27 1’-アルキル修飾リボース誘導体及び使用方法

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Country Status (10)

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

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WO2024081954A2 (en) 2022-10-14 2024-04-18 Sanegene Bio Usa Inc. Small interfering rna targeting c3 and uses thereof
EP4705312A1 (en) * 2023-05-03 2026-03-11 Sanegene Bio USA Inc. Lipid-based conjugates for systemic, central nervous system, peripheral nervous system, and ocular delivery
AU2024301344A1 (en) * 2023-07-24 2026-01-29 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
WO2025143262A1 (ja) 2023-12-28 2025-07-03 日産化学株式会社 カルバモイルエチル修飾を含有する二本鎖rna
TW202608456A (zh) * 2024-05-09 2026-03-01 大陸商石藥集團中奇製藥技術(石家莊)有限公司 GalNAc衍生物及其寡聚核苷酸綴合物
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
WO2025264616A1 (en) * 2024-06-18 2025-12-26 Sanegene Bio Usa Inc. Small interfering rna targeting cfb and uses thereof
WO2026017140A2 (en) 2024-07-18 2026-01-22 Sanegene Bio Usa Inc. Small interfering rna targeting fxi and uses thereof
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
WO2026092658A2 (en) 2024-10-31 2026-05-07 Sanegene Bio Usa Inc. Small interfering rna targeting amyloid beta precursor protein (app) and uses thereof
WO2026102144A2 (en) * 2024-11-06 2026-05-15 Sanegene Bio Usa Inc. Oligonucleotide conjugates of phosphocholine lipid derivatives
US20260226475A1 (en) 2025-01-27 2026-08-06 Sanegene Bio Usa Inc. Small interfering rna targeting acvr1c and uses thereof

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Also Published As

Publication number Publication date
US20260021188A1 (en) 2026-01-22
WO2023014938A1 (en) 2023-02-09
JP2024529067A (ja) 2024-08-01
EP4380946A1 (en) 2024-06-12
JP2026095525A (ja) 2026-06-11
AU2022324471A1 (en) 2024-02-15
US12465647B2 (en) 2025-11-11
US20230138928A1 (en) 2023-05-04
IL310547A (en) 2024-03-01
KR20240070519A (ko) 2024-05-21
CA3227661A1 (en) 2023-02-09
CN117980315A (zh) 2024-05-03
MX2024001581A (es) 2024-04-18

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