JP6561061B2 - ＮＯＴＣＨ １特異的なｓｉＲＮＡ分子 - Google Patents

ＮＯＴＣＨ １特異的なｓｉＲＮＡ分子 Download PDF

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JP6561061B2
JP6561061B2 JP2016544736A JP2016544736A JP6561061B2 JP 6561061 B2 JP6561061 B2 JP 6561061B2 JP 2016544736 A JP2016544736 A JP 2016544736A JP 2016544736 A JP2016544736 A JP 2016544736A JP 6561061 B2 JP6561061 B2 JP 6561061B2
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nucleotides
stretch
antisense
strand
nucleic acid
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JP2016533190A5 (enExample
JP2016533190A (ja
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セーレン シュライバー
セーレン シュライバー
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ソルベンティス ゲーエムベーハー
ソルベンティス ゲーエムベーハー
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    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
    • A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • C—CHEMISTRY; METALLURGY
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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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dispersion Chemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Transition And Organic Metals Composition Catalysts For Addition Polymerization (AREA)
JP2016544736A 2013-09-30 2014-09-30 ＮＯＴＣＨ １特異的なｓｉＲＮＡ分子 Expired - Fee Related JP6561061B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP13004722.8 2013-09-30
EP13004722.8A EP2853595A1 (en) 2013-09-30 2013-09-30 NOTCH 1 specific siRNA molecules
PCT/EP2014/002655 WO2015043768A1 (en) 2013-09-30 2014-09-30 Notch 1 specific sirna molecule

Related Child Applications (1)

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JP2019133268A Division JP7086039B2 (ja) 2013-09-30 2019-07-19 ＮＯＴＣＨ １特異的なｓｉＲＮＡ分子

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JP2016533190A JP2016533190A (ja) 2016-10-27
JP2016533190A5 JP2016533190A5 (enExample) 2017-11-09
JP6561061B2 true JP6561061B2 (ja) 2019-08-14

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JP2016544736A Expired - Fee Related JP6561061B2 (ja) 2013-09-30 2014-09-30 ＮＯＴＣＨ １特異的なｓｉＲＮＡ分子
JP2019133268A Expired - Fee Related JP7086039B2 (ja) 2013-09-30 2019-07-19 ＮＯＴＣＨ １特異的なｓｉＲＮＡ分子

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US (2) US9896690B2 (enExample)
EP (2) EP2853595A1 (enExample)
JP (2) JP6561061B2 (enExample)
CN (2) CN105814202B (enExample)
AU (1) AU2014327809B2 (enExample)
CA (1) CA2924788A1 (enExample)
DK (1) DK3052625T3 (enExample)
HR (1) HRP20211525T1 (enExample)
HU (1) HUE056609T2 (enExample)
IL (1) IL244661B (enExample)
LT (1) LT3052625T (enExample)
MX (1) MX377713B (enExample)
RU (1) RU2651493C2 (enExample)
SG (2) SG10201802593WA (enExample)
SI (1) SI3052625T1 (enExample)
WO (1) WO2015043768A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2853595A1 (en) * 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules
US12459984B2 (en) * 2019-12-27 2025-11-04 National University Corporation Kobe University Cancer gene therapy drug
CN112626068B (zh) * 2020-12-21 2021-12-03 徐州医科大学 一种靶向Notch1基因的LncRNA及其应用
CN116790603B (zh) * 2023-08-18 2023-10-31 成都中科奥格生物科技有限公司 一种sgRNA、CRISPR/Cas9载体及其构建方法和用途

Family Cites Families (19)

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US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
JPH07509133A (ja) 1992-07-17 1995-10-12 リボザイム・ファーマシューティカルズ・インコーポレイテッド 動物疾患の処置のための方法および剤
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US6001311A (en) 1997-02-05 1999-12-14 Protogene Laboratories, Inc. Apparatus for diverse chemical synthesis using two-dimensional array
US6395713B1 (en) 1997-07-23 2002-05-28 Ribozyme Pharmaceuticals, Inc. Compositions for the delivery of negatively charged molecules
CA2326823A1 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
WO2000053722A2 (en) 1999-03-10 2000-09-14 Phogen Limited Delivery of nucleic acids and proteins to cells
EP1857547B2 (en) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Further novel forms of interfering RNA molecules
DK1771206T3 (en) 2004-05-05 2018-05-22 Silence Therapeutics Gmbh LIPIDS, LIPID COMPLEXES AND USE THEREOF
WO2009095517A2 (es) * 2008-01-29 2009-08-06 Proyecto De Biomedicina Cima, S.L. Métodos y composiciones capaces de provocar un silenciamiento post-transcripcional de la expresión génica de forma sinérgica
EP2250265A1 (en) * 2008-03-12 2010-11-17 Intradigm Corporation Compositions comprising notch1 sirna and methods of use thereof
EP2123256A1 (en) * 2008-05-19 2009-11-25 Ruhr-Universität Bochum Perfluorcarbon nanoemulsions with endocytosis enhancing surface for gene-transfer
NZ597504A (en) * 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
WO2013020097A1 (en) * 2011-08-03 2013-02-07 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide compounds for treating hearing and balance disorders
EP2802657B1 (en) * 2012-01-12 2018-05-02 Quark Pharmaceuticals, Inc. Combination therapy for treating hearing and balance disorders
EP2853595A1 (en) * 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules

Also Published As

Publication number Publication date
JP2019196384A (ja) 2019-11-14
CN105814202A (zh) 2016-07-27
EP2853595A1 (en) 2015-04-01
HRP20211525T1 (hr) 2021-12-24
EP3052625B1 (en) 2021-08-25
AU2014327809B2 (en) 2021-11-18
SG10201802593WA (en) 2018-05-30
CA2924788A1 (en) 2015-04-02
US20160215292A1 (en) 2016-07-28
WO2015043768A8 (en) 2016-05-12
IL244661A0 (en) 2016-04-21
MX2016004142A (es) 2016-10-13
DK3052625T3 (da) 2021-11-15
JP2016533190A (ja) 2016-10-27
US10669548B2 (en) 2020-06-02
SI3052625T1 (sl) 2021-12-31
RU2651493C2 (ru) 2018-04-19
IL244661B (en) 2021-08-31
HUE056609T2 (hu) 2022-02-28
US9896690B2 (en) 2018-02-20
WO2015043768A1 (en) 2015-04-02
LT3052625T (lt) 2021-12-10
CN111197046A (zh) 2020-05-26
US20180237785A1 (en) 2018-08-23
RU2016116931A (ru) 2017-11-10
MX377713B (es) 2025-03-11
CN105814202B (zh) 2020-03-06
AU2014327809A1 (en) 2016-04-07
EP3052625A1 (en) 2016-08-10
SG11201602437VA (en) 2016-04-28
JP7086039B2 (ja) 2022-06-17

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