KR20240082361A - 올리고뉴클레오티드의 생체내 전달에 사용하기 위한 2'-알킬 또는 3'-알킬 변형된 리보스 유도체 - Google Patents

올리고뉴클레오티드의 생체내 전달에 사용하기 위한 2'-알킬 또는 3'-알킬 변형된 리보스 유도체 Download PDF

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KR20240082361A
KR20240082361A KR1020247013107A KR20247013107A KR20240082361A KR 20240082361 A KR20240082361 A KR 20240082361A KR 1020247013107 A KR1020247013107 A KR 1020247013107A KR 20247013107 A KR20247013107 A KR 20247013107A KR 20240082361 A KR20240082361 A KR 20240082361A
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scaffold
compound
conjugate
nucleic acid
optionally substituted
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Korean (ko)
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웨이민 왕
시아오촨 카이
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사네진 바이오 유에스에이 인크.
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds 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

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020247013107A 2021-09-22 2022-09-22 올리고뉴클레오티드의 생체내 전달에 사용하기 위한 2'-알킬 또는 3'-알킬 변형된 리보스 유도체 Pending KR20240082361A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163246870P 2021-09-22 2021-09-22
US63/246,870 2021-09-22
PCT/US2022/044377 WO2023049258A1 (en) 2021-09-22 2022-09-22 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides

Publications (1)

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KR20240082361A true KR20240082361A (ko) 2024-06-10

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KR1020247013107A Pending KR20240082361A (ko) 2021-09-22 2022-09-22 올리고뉴클레오티드의 생체내 전달에 사용하기 위한 2'-알킬 또는 3'-알킬 변형된 리보스 유도체

Country Status (9)

Country Link
US (1) US20230241224A1 (https=)
EP (1) EP4405368A1 (https=)
JP (1) JP2024534502A (https=)
KR (1) KR20240082361A (https=)
CN (1) CN118076619A (https=)
AU (1) AU2022352676A1 (https=)
CA (1) CA3233113A1 (https=)
IL (1) IL311486A (https=)
WO (1) WO2023049258A1 (https=)

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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
WO2025144872A2 (en) * 2023-12-29 2025-07-03 Adarx Pharmaceuticals, Inc. Nucleoside-derived lipids for drug delivery
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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CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
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KR102350647B1 (ko) 2016-09-02 2022-01-14 다이서나 파마수이티컬, 인크. 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
WO2021067744A1 (en) * 2019-10-02 2021-04-08 Dicerna Pharmaceuticals, Inc. Chemical modifications of small interfering rna with minimal fluorine content

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JP2024534502A (ja) 2024-09-20
CA3233113A1 (en) 2023-03-30
CN118076619A (zh) 2024-05-24
WO2023049258A1 (en) 2023-03-30
AU2022352676A1 (en) 2024-03-14
IL311486A (en) 2024-05-01
US20230241224A1 (en) 2023-08-03
EP4405368A1 (en) 2024-07-31

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