AU2014327809B2 - Notch 1 specific sirna molecules - Google Patents

Notch 1 specific sirna molecules Download PDF

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Publication number
AU2014327809B2
AU2014327809B2 AU2014327809A AU2014327809A AU2014327809B2 AU 2014327809 B2 AU2014327809 B2 AU 2014327809B2 AU 2014327809 A AU2014327809 A AU 2014327809A AU 2014327809 A AU2014327809 A AU 2014327809A AU 2014327809 B2 AU2014327809 B2 AU 2014327809B2
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Australia
Prior art keywords
nucleotides
nucleic acid
stretch
modified
acid molecule
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Ceased
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AU2014327809A
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AU2014327809A1 (en
Inventor
Soren Schreiber
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Soluventis GmbH
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Soluventis GmbH
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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
    • C12N15/1135—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
    • 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
    • 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
    • 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
    • A61P35/02—Antineoplastic agents specific for leukemia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/1138—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
    • 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
    • C12N2310/10—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
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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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (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)
AU2014327809A 2013-09-30 2014-09-30 Notch 1 specific sirna molecules Ceased AU2014327809B2 (en)

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

Publications (2)

Publication Number Publication Date
AU2014327809A1 AU2014327809A1 (en) 2016-04-07
AU2014327809B2 true AU2014327809B2 (en) 2021-11-18

Family

ID=49304652

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2014327809A Ceased AU2014327809B2 (en) 2013-09-30 2014-09-30 Notch 1 specific sirna molecules

Country Status (16)

Country Link
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
CN115151644B (zh) * 2019-12-27 2024-06-21 国立大学法人神户大学 癌基因治疗药
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013020097A1 (en) * 2011-08-03 2013-02-07 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide compounds for treating hearing and balance disorders
WO2013106494A1 (en) * 2012-01-12 2013-07-18 Quark Pharmaceuticals, Inc. Combination therapy for treating hearing and balance disorders

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DK1695979T3 (da) 1991-12-24 2011-10-10 Isis Pharmaceuticals Inc Gappede modificerede oligonukleotider
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
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
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
EP1071753A2 (en) 1998-04-20 2001-01-31 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
EP1159441B8 (en) 1999-03-10 2008-10-29 Marie Curie Cancer Care Delivery of nucleic acids and proteins to cells
ES2280826T5 (es) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Nuevas formas adicionales de moléculas de ARN de interferencia
CA2565633C (en) 2004-05-05 2016-04-26 Atugen Ag Lipids, lipid complexes and use thereof
BRPI0906657A2 (pt) * 2008-01-29 2015-07-14 Proyecto Biomedicina Cima Sl Métodos e composições capazes de causar um silenciamento pós-transcricional da expressão genética de forma sinérgica
CA2717496A1 (en) * 2008-03-12 2009-09-17 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
EP2442792A4 (en) * 2009-06-15 2015-12-23 Alnylam Pharmaceuticals Inc TESTING GENE PCSK9 LIPID-FORMULATED DSRNA
EP2853595A1 (en) * 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013020097A1 (en) * 2011-08-03 2013-02-07 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide compounds for treating hearing and balance disorders
WO2013106494A1 (en) * 2012-01-12 2013-07-18 Quark Pharmaceuticals, Inc. Combination therapy for treating hearing and balance disorders

Also Published As

Publication number Publication date
AU2014327809A1 (en) 2016-04-07
RU2016116931A (ru) 2017-11-10
US10669548B2 (en) 2020-06-02
JP6561061B2 (ja) 2019-08-14
HRP20211525T1 (hr) 2021-12-24
CA2924788A1 (en) 2015-04-02
WO2015043768A1 (en) 2015-04-02
EP2853595A1 (en) 2015-04-01
SG10201802593WA (en) 2018-05-30
MX377713B (es) 2025-03-11
US9896690B2 (en) 2018-02-20
DK3052625T3 (da) 2021-11-15
WO2015043768A8 (en) 2016-05-12
HUE056609T2 (hu) 2022-02-28
SI3052625T1 (sl) 2021-12-31
US20180237785A1 (en) 2018-08-23
LT3052625T (lt) 2021-12-10
CN111197046A (zh) 2020-05-26
SG11201602437VA (en) 2016-04-28
EP3052625A1 (en) 2016-08-10
CN105814202B (zh) 2020-03-06
JP2016533190A (ja) 2016-10-27
MX2016004142A (es) 2016-10-13
CN105814202A (zh) 2016-07-27
EP3052625B1 (en) 2021-08-25
JP7086039B2 (ja) 2022-06-17
RU2651493C2 (ru) 2018-04-19
IL244661B (en) 2021-08-31
IL244661A0 (en) 2016-04-21
US20160215292A1 (en) 2016-07-28
JP2019196384A (ja) 2019-11-14

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Date Code Title Description
DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS: AMEND THE INVENTION TITLE TO READ NOTCH 1 SPECIFIC SIRNA MOLECULES

FGA Letters patent sealed or granted (standard patent)
MK14 Patent ceased section 143(a) (annual fees not paid) or expired