CN114555621B - 键修饰的寡聚化合物及其用途 - Google Patents

键修饰的寡聚化合物及其用途

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Publication number
CN114555621B
CN114555621B CN202080072321.6A CN202080072321A CN114555621B CN 114555621 B CN114555621 B CN 114555621B CN 202080072321 A CN202080072321 A CN 202080072321A CN 114555621 B CN114555621 B CN 114555621B
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Prior art keywords
oligomeric compound
modified oligonucleotide
region
nucleoside
substituted
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CN202080072321.6A
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Chinese (zh)
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CN114555621A (zh
Inventor
梁雪海
P·P·塞斯
B·A·安德森
W·J·德鲁里三世
M·欧斯迦德
M·T·米伽瓦
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Priority to CN202511437623.XA priority Critical patent/CN121779475A/zh
Publication of CN114555621A publication Critical patent/CN114555621A/zh
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    • 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
    • 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
    • A61K31/01—Hydrocarbons
    • A61K31/015—Hydrocarbons carbocyclic
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7048—Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin, digitoxin or digoxin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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    • 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
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    • 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 ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
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    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN202080072321.6A 2019-08-15 2020-08-14 键修饰的寡聚化合物及其用途 Active CN114555621B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202511437623.XA CN121779475A (zh) 2019-08-15 2020-08-14 键修饰的寡聚化合物及其用途

Applications Claiming Priority (9)

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US201962887525P 2019-08-15 2019-08-15
US62/887,525 2019-08-15
US201962953121P 2019-12-23 2019-12-23
US62/953,121 2019-12-23
US202062989442P 2020-03-13 2020-03-13
US62/989,442 2020-03-13
US202063050042P 2020-07-09 2020-07-09
US63/050,042 2020-07-09
PCT/US2020/046561 WO2021030778A1 (en) 2019-08-15 2020-08-14 Linkage modified oligomeric compounds and uses thereof

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CN114555621B true CN114555621B (zh) 2025-10-28

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US (3) US11629348B2 (https=)
EP (1) EP4013767A4 (https=)
JP (2) JP7803850B2 (https=)
KR (1) KR20220062517A (https=)
CN (2) CN114555621B (https=)
WO (1) WO2021030778A1 (https=)

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JP2026012736A (ja) 2026-01-27
CN114555621A (zh) 2022-05-27
EP4013767A1 (en) 2022-06-22
US12595482B2 (en) 2026-04-07
JP7803850B2 (ja) 2026-01-21
US20240002851A1 (en) 2024-01-04
US11629348B2 (en) 2023-04-18
US20220186222A1 (en) 2022-06-16
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US20250368995A1 (en) 2025-12-04

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