JPWO2020184700A1 - - Google Patents

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Publication number
JPWO2020184700A1
JPWO2020184700A1 JP2021505155A JP2021505155A JPWO2020184700A1 JP WO2020184700 A1 JPWO2020184700 A1 JP WO2020184700A1 JP 2021505155 A JP2021505155 A JP 2021505155A JP 2021505155 A JP2021505155 A JP 2021505155A JP WO2020184700 A1 JPWO2020184700 A1 JP WO2020184700A1
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JP
Japan
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JP2021505155A
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JP7530106B2 (ja
JPWO2020184700A5 (https=
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • 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
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3517Marker; Tag
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
  • Microbiology (AREA)
  • Pulmonology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • 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)

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

Family

ID=72427147

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021505155A Active JP7530106B2 (ja) 2019-03-14 2020-03-13 Ihh発現を調節するための核酸複合体

Country Status (4)

Country Link
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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JP3781879B2 (ja) 1996-11-18 2006-05-31 武 今西 新規ヌクレオチド類縁体
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
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
WO2009138516A1 (en) * 2008-05-16 2009-11-19 Galderma Research & Development Therapy regimen for treating acne related diseases
WO2011005765A1 (en) 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Bioprocessing
EP2295543A1 (en) * 2009-09-11 2011-03-16 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Method for the preparation of an influenza virus
WO2012096545A2 (ko) * 2011-01-13 2012-07-19 연세대학교 산학협력단 췌장암 암줄기세포 특성을 이용한 췌장암 신규 바이오마커 및 그의 용도
KR101870915B1 (ko) * 2011-04-08 2018-06-25 유니버시티 오브 레스터 Masp-2 의존적 보체 활성화와 관련된 질병을 치료하는 방법
KR20140128257A (ko) * 2013-04-26 2014-11-05 주식회사 에이앤알티 표적 단백질 스크리닝을 위한 수용성 수용체 라이브러리 및 이를 이용한 표적 단백질 스크리닝 방법
KR20260048600A (ko) * 2015-03-27 2026-04-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 변형된 t 세포 및 이의 제조 및 사용 방법
WO2017131124A1 (ja) 2016-01-26 2017-08-03 日産化学工業株式会社 一本鎖オリゴヌクレオチド
WO2017184586A1 (en) * 2016-04-18 2017-10-26 The Trustees Of Columbia University In The City Of New York Therapeutic targets involved in the progression of nonalcoholic steatohepatitis (nash)
CN108728440A (zh) * 2017-04-25 2018-11-02 上海吉倍生物技术有限公司 Ctla-4基因的用途及相关药物
JP2019047703A (ja) 2017-09-07 2019-03-22 イビデン株式会社 モータ用コイル

Patent 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
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

Also Published As

Publication number Publication date
JP7530106B2 (ja) 2024-08-07
US20220162607A1 (en) 2022-05-26
US12492399B2 (en) 2025-12-09
EP3981431A4 (en) 2023-06-28
EP3981431A1 (en) 2022-04-13
WO2020184700A1 (ja) 2020-09-17

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