JP2024527584A - ＣＮＳ送達のためのＢｉｓ－ＲＮＡｉ化合物 - Google Patents

ＣＮＳ送達のためのＢｉｓ－ＲＮＡｉ化合物 Download PDF

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JP2024527584A
JP2024527584A JP2024500514A JP2024500514A JP2024527584A JP 2024527584 A JP2024527584 A JP 2024527584A JP 2024500514 A JP2024500514 A JP 2024500514A JP 2024500514 A JP2024500514 A JP 2024500514A JP 2024527584 A JP2024527584 A JP 2024527584A
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nucleic acid
linker
strand
nucleotides
dsrna molecule
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Japanese (ja)
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JP2024527584A5 (https=
Inventor
ズラテブ，アイバン
エス サイル，クリストファー
マノハラン，ムティア
ピー レンティーニ，スコット
成夫 松田
ケイ ナイル，ジャヤプラカシュ
ペン，ハイヤン
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of JP2024527584A publication Critical patent/JP2024527584A/ja
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    • 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
    • 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
    • A61K31/658—Medicinal preparations containing organic active ingredients o-phenolic cannabinoids, e.g. cannabidiol, cannabigerolic acid, cannabichromene or tetrahydrocannabinol
    • 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
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • 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
    • C12N15/1137—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 enzymes
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    • 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]
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Biomedical Technology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
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  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2024500514A 2021-07-09 2022-07-08 ＣＮＳ送達のためのＢｉｓ－ＲＮＡｉ化合物 Pending JP2024527584A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163219930P 2021-07-09 2021-07-09
US202163220232P 2021-07-09 2021-07-09
US63/219,930 2021-07-09
US63/220,232 2021-07-09
PCT/US2022/036455 WO2023283403A2 (en) 2021-07-09 2022-07-08 Bis-rnai compounds for cns delivery

Publications (2)

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JP2024527584A true JP2024527584A (ja) 2024-07-25
JP2024527584A5 JP2024527584A5 (https=) 2025-07-15

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JP2024500514A Pending JP2024527584A (ja) 2021-07-09 2022-07-08 ＣＮＳ送達のためのＢｉｓ－ＲＮＡｉ化合物

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US (1) US20250270562A1 (https=)
EP (1) EP4367237A2 (https=)
JP (1) JP2024527584A (https=)
WO (1) WO2023283403A2 (https=)

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Publication number Priority date Publication date Assignee Title
CN121752721A (zh) * 2023-05-31 2026-03-27 箭头药业股份有限公司 用于多聚体RNAi剂缀合物的肝脏递送平台及其使用方法
WO2025066936A1 (zh) * 2023-09-27 2025-04-03 北京炫景瑞医药科技有限公司 包含多个双链寡核苷酸分子的核酸嵌合体、组合物及其用途
WO2025157273A1 (en) * 2024-01-25 2025-07-31 Shanghai Rona Therapeutics Co., Ltd. Two or more nucleic acid molecules connected by a linker
WO2025223315A1 (zh) * 2024-04-25 2025-10-30 苏州时安生物技术有限公司 一种用于肝脏特异性递送的多靶标双链rna偶联物及药物组合物
WO2025247382A1 (zh) * 2024-05-30 2025-12-04 北京超环生物科技有限公司 用于基因沉默的双链环状siRNA组装体
WO2025247332A1 (zh) * 2024-05-31 2025-12-04 石药集团中奇制药技术（石家庄）有限公司 一种抑制vegfa基因和ang-2基因表达的双靶核酸分子

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