JP7844347B2 - アポリポタンパク質Ｅ（ＡｐｏＥ）ｉＲＮＡ剤組成物およびその使用方法 - Google Patents

アポリポタンパク質Ｅ（ＡｐｏＥ）ｉＲＮＡ剤組成物およびその使用方法

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Publication number
JP7844347B2
JP7844347B2 JP2022565879A JP2022565879A JP7844347B2 JP 7844347 B2 JP7844347 B2 JP 7844347B2 JP 2022565879 A JP2022565879 A JP 2022565879A JP 2022565879 A JP2022565879 A JP 2022565879A JP 7844347 B2 JP7844347 B2 JP 7844347B2
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nucleotides
nucleotide
apoe
strand
antisense strand
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Japanese (ja)
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JP2023523993A (ja
JP2023523993A5 (enExample
Inventor
リー ボストウィック，ブレット
ペン，ハイヤン
ディ マキニンチ，ジェイムズ
カストレノ，アダム
ケイ シュリーゲル，マーク
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/30—Chemical structure
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
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    • C12N2320/31—Combination therapy
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Steroid Compounds (AREA)
JP2022565879A 2020-04-27 2021-04-26 アポリポタンパク質Ｅ（ＡｐｏＥ）ｉＲＮＡ剤組成物およびその使用方法 Active JP7844347B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063015867P 2020-04-27 2020-04-27
US63/015,867 2020-04-27
PCT/US2021/029081 WO2021222065A1 (en) 2020-04-27 2021-04-26 Apolipoprotein e (apoe) irna agent compositions and methods of use thereof

Publications (3)

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JP2023523993A JP2023523993A (ja) 2023-06-08
JP2023523993A5 JP2023523993A5 (enExample) 2025-04-30
JP7844347B2 true JP7844347B2 (ja) 2026-04-13

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Country Link
US (1) US20230193268A1 (enExample)
EP (1) EP4143319A1 (enExample)
JP (1) JP7844347B2 (enExample)
KR (1) KR20230018377A (enExample)
CN (1) CN115955972A (enExample)
AU (1) AU2021263554A1 (enExample)
BR (1) BR112022021813A2 (enExample)
CA (1) CA3181400A1 (enExample)
IL (1) IL297702A (enExample)
MX (1) MX2022013525A (enExample)
WO (1) WO2021222065A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4216964A4 (en) * 2020-09-24 2025-02-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing apoe expression
TW202328449A (zh) 2021-09-24 2023-07-16 美商艾拉倫製藥公司 微管相關蛋白ＴＡＵ（ＭＡＰＴ）ｉＲＮＡ試劑組合物及其使用方法
CN118318042A (zh) * 2021-11-03 2024-07-09 豪夫迈·罗氏有限公司 用于调节载脂蛋白e4表达的寡核苷酸
TW202335674A (zh) * 2021-11-05 2023-09-16 美商黛瑟納製藥公司 靶定中樞神經系統的星狀細胞之脂質結合物
EP4466357A2 (en) * 2022-01-23 2024-11-27 Regeneron Pharmaceuticals, Inc. Methods of improving health with apolipoprotein e (apoe) inhibitors
CN120189530A (zh) * 2023-11-22 2025-06-24 广州瑞风生物科技有限公司 基因编辑系统及其应用
WO2025159427A1 (ko) * 2024-01-26 2025-07-31 서울대학교산학협력단 신규한 apoe 안티센스 올리고뉴클레오타이드 및 이의 용도

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof

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