KR20240161967A - 트랜스페린 수용체 2 의 발현을 억제하는 siRNA - Google Patents

트랜스페린 수용체 2 의 발현을 억제하는 siRNA Download PDF

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KR20240161967A
KR20240161967A KR1020247034184A KR20247034184A KR20240161967A KR 20240161967 A KR20240161967 A KR 20240161967A KR 1020247034184 A KR1020247034184 A KR 1020247034184A KR 20247034184 A KR20247034184 A KR 20247034184A KR 20240161967 A KR20240161967 A KR 20240161967A
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rna
oligonucleotide
nucleoside
pharmaceutically acceptable
acceptable salt
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Korean (ko)
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마코토 고이즈미
다카오 쇼지
다이치 후쿠나가
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다이이찌 산쿄 가부시키가이샤
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    • A—HUMAN NECESSITIES
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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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • 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
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    • A—HUMAN NECESSITIES
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    • 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
    • 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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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KR1020247034184A 2022-03-16 2023-03-15 트랜스페린 수용체 2 의 발현을 억제하는 siRNA Pending KR20240161967A (ko)

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JP2022041864 2022-03-16
JPJP-P-2022-041864 2022-03-16
PCT/JP2023/009973 WO2023176862A1 (ja) 2022-03-16 2023-03-15 トランスフェリン受容体２の発現を抑制するｓｉＲＮＡ

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US (1) US20250188478A1 (https=)
EP (1) EP4495240A1 (https=)
JP (1) JPWO2023176862A1 (https=)
KR (1) KR20240161967A (https=)
CN (1) CN118891366A (https=)
AU (1) AU2023234185A1 (https=)
CO (1) CO2024013601A2 (https=)
IL (1) IL315659A (https=)
MX (1) MX2024011244A (https=)
TW (1) TW202345867A (https=)
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WO2026052078A1 (zh) * 2024-09-07 2026-03-12 苏州瑞博生物技术股份有限公司 寡核苷酸、寡核苷酸缀合物及组合物和用途
US12454693B1 (en) * 2024-09-27 2025-10-28 Dexcel Pharma Technologies Ltd. Nucleic acids for inhibiting expression of transferrin receptor 2

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US20250188478A1 (en) 2025-06-12
TW202345867A (zh) 2023-12-01
JPWO2023176862A1 (https=) 2023-09-21
CO2024013601A2 (es) 2024-10-31
IL315659A (en) 2024-11-01
WO2023176862A1 (ja) 2023-09-21
CN118891366A (zh) 2024-11-01
EP4495240A1 (en) 2025-01-22
AU2023234185A1 (en) 2024-10-03

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