JP7530106B2 - Ｉｈｈ発現を調節するための核酸複合体 - Google Patents

Ｉｈｈ発現を調節するための核酸複合体 Download PDF

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JP7530106B2
JP7530106B2 JP2021505155A JP2021505155A JP7530106B2 JP 7530106 B2 JP7530106 B2 JP 7530106B2 JP 2021505155 A JP2021505155 A JP 2021505155A JP 2021505155 A JP2021505155 A JP 2021505155A JP 7530106 B2 JP7530106 B2 JP 7530106B2
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nucleic acid
acid complex
oligonucleotide
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ihh
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JPWO2020184700A5 (https=
JPWO2020184700A1 (https=
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秀樹 木澤
鋼 高木
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Rena Therapeutics Inc
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Rena Therapeutics Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
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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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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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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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    • C12N2310/3517—Marker; Tag
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021505155A 2019-03-14 2020-03-13 Ｉｈｈ発現を調節するための核酸複合体 Active JP7530106B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019047703 2019-03-14
JP2019047703 2019-03-14
PCT/JP2020/011018 WO2020184700A1 (ja) 2019-03-14 2020-03-13 Ｉｈｈ発現を調節するための核酸複合体

Publications (3)

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JPWO2020184700A1 JPWO2020184700A1 (https=) 2020-09-17
JPWO2020184700A5 JPWO2020184700A5 (https=) 2023-02-27
JP7530106B2 true JP7530106B2 (ja) 2024-08-07

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US (1) US12492399B2 (https=)
EP (1) EP3981431A4 (https=)
JP (1) JP7530106B2 (https=)
WO (1) WO2020184700A1 (https=)

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007530571A (ja) 2004-03-26 2007-11-01 キュリス インコーポレイテッド ヘッジホッグシグナリングのｒｎａ干渉モジュレーター及びその利用
JP2008512087A (ja) 2004-06-17 2008-04-24 スラソス セラピューティックス インコーポレーテッド Ｔｄｆ関連化合物およびその類似体
US20110183948A1 (en) 2010-01-15 2011-07-28 Infinity Pharmaceuticals, Inc. Treatment of fibrotic conditions using hedgehog inhibitors
US20120252870A1 (en) 2011-02-19 2012-10-04 Rhode Island Hospital Indian Hedgehog (Ihh) as a Marker to Predict Osteoarthritis (OA) and Methods for the Prevention and Treatment of OA
JP2013099338A (ja) 2008-08-25 2013-05-23 Excaliard Pharmaceuticals Inc 結合組織成長因子を対象とするアンチセンスオリゴヌクレオチドおよびその使用
JP2014511694A (ja) 2011-04-03 2014-05-19 ザ ジェネラル ホスピタル コーポレーション ドゥーイング ビジネス アズ マサチューセッツ ジェネラル ホスピタル 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現
JP2014530004A (ja) 2011-09-20 2014-11-17 アイシス ファーマシューティカルズ， インコーポレーテッド Ｇｃｇｒ発現のアンチセンス調整
WO2019022196A1 (ja) 2017-07-26 2019-01-31 日産化学株式会社 一本鎖オリゴヌクレオチド

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AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
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Publication number Priority date Publication date Assignee Title
JP2007530571A (ja) 2004-03-26 2007-11-01 キュリス インコーポレイテッド ヘッジホッグシグナリングのｒｎａ干渉モジュレーター及びその利用
JP2008512087A (ja) 2004-06-17 2008-04-24 スラソス セラピューティックス インコーポレーテッド Ｔｄｆ関連化合物およびその類似体
JP2013099338A (ja) 2008-08-25 2013-05-23 Excaliard Pharmaceuticals Inc 結合組織成長因子を対象とするアンチセンスオリゴヌクレオチドおよびその使用
US20110183948A1 (en) 2010-01-15 2011-07-28 Infinity Pharmaceuticals, Inc. Treatment of fibrotic conditions using hedgehog inhibitors
US20120252870A1 (en) 2011-02-19 2012-10-04 Rhode Island Hospital Indian Hedgehog (Ihh) as a Marker to Predict Osteoarthritis (OA) and Methods for the Prevention and Treatment of OA
JP2014511694A (ja) 2011-04-03 2014-05-19 ザ ジェネラル ホスピタル コーポレーション ドゥーイング ビジネス アズ マサチューセッツ ジェネラル ホスピタル 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現
JP2014530004A (ja) 2011-09-20 2014-11-17 アイシス ファーマシューティカルズ， インコーポレーテッド Ｇｃｇｒ発現のアンチセンス調整
WO2019022196A1 (ja) 2017-07-26 2019-01-31 日産化学株式会社 一本鎖オリゴヌクレオチド

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US20220162607A1 (en) 2022-05-26
US12492399B2 (en) 2025-12-09
EP3981431A4 (en) 2023-06-28
JPWO2020184700A1 (https=) 2020-09-17
EP3981431A1 (en) 2022-04-13
WO2020184700A1 (ja) 2020-09-17

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