JP7262816B2 - ヘテロ核酸のbbb通過脂質リガンド - Google Patents

ヘテロ核酸のbbb通過脂質リガンド Download PDF

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JP7262816B2
JP7262816B2 JP2020507927A JP2020507927A JP7262816B2 JP 7262816 B2 JP7262816 B2 JP 7262816B2 JP 2020507927 A JP2020507927 A JP 2020507927A JP 2020507927 A JP2020507927 A JP 2020507927A JP 7262816 B2 JP7262816 B2 JP 7262816B2
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nucleic acid
malat1
strand
crna
oligonucleotide
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JPWO2019182109A1 (ja
JPWO2019182109A5 (https=
Inventor
隆徳 横田
哲也 永田
英紀 古川
泰男 中川
能紀 余郷
僚資 得能
茂和 佐々木
功介 日▲高▼
倫宏 関
健一 宮田
祐介 安達
直人 有村
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Tokyo Medical and Dental University NUC
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Tokyo Medical and Dental University NUC
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Publication of JPWO2019182109A1 publication Critical patent/JPWO2019182109A1/ja
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Priority to JP2023061429A priority Critical patent/JP7669602B2/ja
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Priority to JP2024188699A priority patent/JP2025063018A/ja
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/542Carboxylic acids, e.g. a fatty acid or an amino acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/55Medicinal 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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
    • A61K47/551Medicinal 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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug one of the codrug's components being a vitamin, e.g. niacinamide, vitamin B3, cobalamin, vitamin B12, folate, vitamin A or retinoic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/51Medicinal 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
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    • A61K47/554Medicinal 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 the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
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    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/31Chemical structure of the backbone
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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JP2020507927A 2018-03-22 2019-03-22 ヘテロ核酸のbbb通過脂質リガンド Active JP7262816B2 (ja)

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JP2023061429A JP7669602B2 (ja) 2018-03-22 2023-04-05 ヘテロ核酸のbbb通過脂質リガンド
JP2024188699A JP2025063018A (ja) 2018-03-22 2024-10-28 ヘテロ核酸のbbb通過脂質リガンド

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JP2018055372 2018-03-22
JP2018055372 2018-03-22
PCT/JP2019/012077 WO2019182109A1 (ja) 2018-03-22 2019-03-22 ヘテロ核酸のbbb通過脂質リガンド

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JPWO2019182109A1 JPWO2019182109A1 (ja) 2021-03-11
JPWO2019182109A5 JPWO2019182109A5 (https=) 2022-03-24
JP7262816B2 true JP7262816B2 (ja) 2023-04-24

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JP2020507927A Active JP7262816B2 (ja) 2018-03-22 2019-03-22 ヘテロ核酸のbbb通過脂質リガンド
JP2023061429A Active JP7669602B2 (ja) 2018-03-22 2023-04-05 ヘテロ核酸のbbb通過脂質リガンド
JP2024188699A Pending JP2025063018A (ja) 2018-03-22 2024-10-28 ヘテロ核酸のbbb通過脂質リガンド

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JP2024188699A Pending JP2025063018A (ja) 2018-03-22 2024-10-28 ヘテロ核酸のbbb通過脂質リガンド

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US (1) US20250333736A1 (https=)
EP (1) EP3770257B1 (https=)
JP (3) JP7262816B2 (https=)
WO (1) WO2019182109A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023080164A (ja) * 2018-03-22 2023-06-08 国立大学法人 東京医科歯科大学 ヘテロ核酸のbbb通過脂質リガンド

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3766972B1 (en) * 2018-03-14 2025-04-23 Institute of Science Tokyo Nucleic acid complex
US20220370491A1 (en) * 2019-09-18 2022-11-24 National University Corporation Tokyo Medical And Dental University Nucleic acid complex
WO2022106695A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
WO2023042876A1 (ja) * 2021-09-15 2023-03-23 国立大学法人東京医科歯科大学 2'-修飾ヌクレオシドを含むヘテロ核酸
CN116789726A (zh) * 2022-01-28 2023-09-22 珂阑(上海)医药科技有限公司 甾类化合物、其制备方法和应用
JPWO2024071099A1 (https=) 2022-09-29 2024-04-04
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015502134A (ja) 2011-12-16 2015-01-22 国立大学法人 東京医科歯科大学 キメラ2重鎖核酸
JP2015527314A (ja) 2012-07-10 2015-09-17 ベースクリック ゲーエムベーハー アナンダミド−修飾核酸分子
WO2017053999A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2018056442A1 (ja) 2016-09-23 2018-03-29 国立大学法人東京医科歯科大学 血液脳関門通過型ヘテロ2本鎖核酸
WO2018062510A1 (ja) 2016-09-29 2018-04-05 国立大学法人東京医科歯科大学 オーバーハングを有する二本鎖核酸複合体

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4912873B2 (ja) * 2003-04-09 2012-04-11 アルナイラム ファーマシューティカルズ, インコーポレイテッド iRNA複合体
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
US20080039415A1 (en) * 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
ES2603379T3 (es) 2006-10-09 2017-02-27 Roche Innovation Center Copenhagen A/S Compuestos antagonistas de ARN para la modulación de PCSK9
WO2008049085A1 (en) 2006-10-18 2008-04-24 Isis Pharmaceuticals, Inc. Antisense compounds
JP6562515B2 (ja) 2016-09-28 2019-08-21 尾花 敏▲徳▼ セキュリティシステム
JP7262816B2 (ja) 2018-03-22 2023-04-24 国立大学法人 東京医科歯科大学 ヘテロ核酸のbbb通過脂質リガンド

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015502134A (ja) 2011-12-16 2015-01-22 国立大学法人 東京医科歯科大学 キメラ2重鎖核酸
JP2015527314A (ja) 2012-07-10 2015-09-17 ベースクリック ゲーエムベーハー アナンダミド−修飾核酸分子
WO2017053999A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2018056442A1 (ja) 2016-09-23 2018-03-29 国立大学法人東京医科歯科大学 血液脳関門通過型ヘテロ2本鎖核酸
WO2018062510A1 (ja) 2016-09-29 2018-04-05 国立大学法人東京医科歯科大学 オーバーハングを有する二本鎖核酸複合体

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Scientific Reports (2018) Vol.8, No.4377, pp.1-12 (published online 12 March 2018)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023080164A (ja) * 2018-03-22 2023-06-08 国立大学法人 東京医科歯科大学 ヘテロ核酸のbbb通過脂質リガンド
JP7669602B2 (ja) 2018-03-22 2025-04-30 国立大学法人東京科学大学 ヘテロ核酸のbbb通過脂質リガンド

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EP3770257A1 (en) 2021-01-27
EP3770257A4 (en) 2022-03-09
JP2023080164A (ja) 2023-06-08
EP3770257B1 (en) 2026-01-28
JP2025063018A (ja) 2025-04-15
US20250333736A1 (en) 2025-10-30
JPWO2019182109A1 (ja) 2021-03-11
JP7669602B2 (ja) 2025-04-30
WO2019182109A1 (ja) 2019-09-26

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