JP2023543215A - Ａｐｏｅの発現を低減するための化合物及び方法 - Google Patents

Ａｐｏｅの発現を低減するための化合物及び方法 Download PDF

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Publication number
JP2023543215A
JP2023543215A JP2023518754A JP2023518754A JP2023543215A JP 2023543215 A JP2023543215 A JP 2023543215A JP 2023518754 A JP2023518754 A JP 2023518754A JP 2023518754 A JP2023518754 A JP 2023518754A JP 2023543215 A JP2023543215 A JP 2023543215A
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modified
oligonucleotide
certain embodiments
nucleosides
oligomeric
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Pending
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JP2023518754A
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Japanese (ja)
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JP2023543215A5 (https=
Inventor
コール，トレーシー・エイ
ムコパディヤーイ，スワガタム
ブイ，フィン－ホア
シング，プリヤム
コルダシェヴィッチ，ホリー
フレアー，スーザン・エム
チャオ，ヒエン・トゥイ
プラカシュ，タジャ・ピー
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of JP2023543215A publication Critical patent/JP2023543215A/ja
Publication of JP2023543215A5 publication Critical patent/JP2023543215A5/ja
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/26—Endoribonucleases producing 5'-phosphomonoesters (3.1.26)
    • C12Y301/26004—Ribonuclease H (3.1.26.4)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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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]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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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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  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
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JP2023518754A 2020-09-24 2021-09-23 Ａｐｏｅの発現を低減するための化合物及び方法 Pending JP2023543215A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063083006P 2020-09-24 2020-09-24
US63/083,006 2020-09-24
PCT/US2021/051822 WO2022066956A1 (en) 2020-09-24 2021-09-23 Compounds and methods for reducing apoe expression

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JP2023543215A true JP2023543215A (ja) 2023-10-13
JP2023543215A5 JP2023543215A5 (https=) 2024-10-21

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JP2023518754A Pending JP2023543215A (ja) 2020-09-24 2021-09-23 Ａｐｏｅの発現を低減するための化合物及び方法

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US (1) US20230357770A1 (https=)
EP (1) EP4216964A4 (https=)
JP (1) JP2023543215A (https=)
WO (1) WO2022066956A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121013908A (zh) * 2023-02-13 2025-11-25 赛神医药有限公司 调节载脂蛋白e(apoe)表达的寡核苷酸及其使用方法
WO2025240846A1 (en) * 2024-05-17 2025-11-20 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apoe expression

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7517644B1 (en) * 1991-08-23 2009-04-14 Larry J. Smith Method and compositions for cellular reprogramming
CN110257510A (zh) * 2019-08-06 2019-09-20 江苏贝格尔生物医药有限公司 一种检测apoe基因多态性的引物和探针及试剂盒

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006510341A (ja) * 2002-02-27 2006-03-30 バイエル・ヘルスケア・アクチェンゲゼルシャフト 有害薬剤反応および薬剤効力を予測する単一ヌクレオチド多型
US10472634B2 (en) * 2014-06-04 2019-11-12 Ionis Pharmaceuticals, Inc. Antisense compounds targeting apolipoprotein E receptor 2
EP3191578B1 (en) * 2014-09-12 2020-11-04 Whitehead Institute for Biomedical Research Cells expressing apolipoprotein e and uses thereof
ES3039647T3 (en) * 2017-05-31 2025-10-23 Arcturus Therapeutics Inc Synthesis and structure of high potency rna therapeutics
US10517889B2 (en) * 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
AU2020227824B2 (en) * 2019-02-27 2025-07-10 Ionis Pharmaceuticals, Inc. Modulators of MALAT1 expression
EP3997227A1 (en) * 2019-07-10 2022-05-18 Locanabio, Inc. Rna-targeting knockdown and replacement compositions and methods for use
IL297702A (en) * 2020-04-27 2022-12-01 Alnylam Pharmaceuticals Inc Apolipoprotein e (apoe) irna agent compositions and methods of use thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7517644B1 (en) * 1991-08-23 2009-04-14 Larry J. Smith Method and compositions for cellular reprogramming
CN110257510A (zh) * 2019-08-06 2019-09-20 江苏贝格尔生物医药有限公司 一种检测apoe基因多态性的引物和探针及试剂盒

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NEURON, vol. 96, JPN6025040271, 2017, pages 1013 - 1023, ISSN: 0005699574 *

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EP4216964A1 (en) 2023-08-02
EP4216964A4 (en) 2025-02-12
US20230357770A1 (en) 2023-11-09

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