NO20083208L - RNA-interferensmediert inhibering av hepatitt C-virus-genekspresjon ved anvendelse av korte interfererende nukleinsyrer (siNA) - Google Patents

RNA-interferensmediert inhibering av hepatitt C-virus-genekspresjon ved anvendelse av korte interfererende nukleinsyrer (siNA)

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Publication number
NO20083208L
NO20083208L NO20083208A NO20083208A NO20083208L NO 20083208 L NO20083208 L NO 20083208L NO 20083208 A NO20083208 A NO 20083208A NO 20083208 A NO20083208 A NO 20083208A NO 20083208 L NO20083208 L NO 20083208L
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Norway
Prior art keywords
nucleic acid
acid molecules
rna
gene expression
small nucleic
Prior art date
Application number
NO20083208A
Other languages
English (en)
Inventor
James Mcswiggen
David Morrissey
Roberto Guerciolini
Chandra Vargeese
Vasant Jadhav
Original Assignee
Sirna Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from US11/311,826 external-priority patent/US20060211642A1/en
Application filed by Sirna Therapeutics Inc filed Critical Sirna Therapeutics Inc
Publication of NO20083208L publication Critical patent/NO20083208L/no

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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
    • C12N15/1131—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 viruses
    • 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
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • 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
    • A61K9/127—Liposomes
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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 N.A.
    • 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
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/24011—Flaviviridae
    • C12N2770/24211—Hepacivirus, e.g. hepatitis C virus, hepatitis G virus

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Foreliggende oppfinnelse gjelder forbindelser, preparater og fremgangsmåter for undersøkelse, diagnose og behandling av trekk, sykdommer og tilstander som responderer på modulering av genekspresjon og/eller genaktivitet. Foreliggende oppfinnelse er også rettet mot forbindelser, preparater og fremgangsmåter forbundet med trekk, sykdommer og tilstander som responderer på modulering av ekspresjonen og/eller aktiviteten av gener som inngår i genekspresjonsveier eller andre cellulære prosesser som formidler opprettholdelse eller utvikling av slike trekk, sykdommer og tilstander. Nærmere bestemt gjelder oppfinnelsen dobbeltrådete nukleinsyremolekyler, innbefattet små nukleinsyremolekyler, for eksempel kort interfererende nukleinsyre (siNA), kort interfererende RNA (siRNA), dobbeltrådet RNA (dsRNA), mikro-RNA (miRNA) og korte håmåls-RNA (shRNA)molekyler som kan formidle RNA-interferens (RNAi) mot genekspresjon, innbefattet blandinger av slike små nukleinsyremolekyler og lipidnanopartikkel (LNP)-utforminger av slike små nukleinsyremolekyler. Foreliggende oppfinnelse gjelder også små nukleinsyremolekyler, for eksempel siNA, siRNA og andre, som kan inbibere funksjonen av endogene RNA-molekyler, for eksempel endogent mikro-RNA (miRNA) (for eksempel miRNAinhibitorer) eller endogent, kort interfererende RNA (siRNA), (for eksempel siRNA-inhibitorer), eller som kan inhibere funksjonen av RISC (for eksempel RISC-inhibitorer), for modulering av genekspresjon ved å interferere med den regulatoriske funksjon av slike endogene RNA eller proteiner forbundet med slike endogene RNA (for eksempel RISC) innbefattet blandinger av slike små nukleinsyremolekyler og lipidnanopartikkel (LNP)-utforminger av slike små nukleinsyremolekyler. Slike små nukleinsyremolekyler er for eksempel anvendbare for oppnåelse av preparater for forebyggelse, inhibering eller reduksjon av sykdommer, trekk og tilstander som er forbundet med genekspresj on eller genaktivitet i et individ eller en organisme.
NO20083208A 2005-12-19 2008-07-18 RNA-interferensmediert inhibering av hepatitt C-virus-genekspresjon ved anvendelse av korte interfererende nukleinsyrer (siNA) NO20083208L (no)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11/311,826 US20060211642A1 (en) 2001-05-18 2005-12-19 RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)
US11/510,872 US20080207542A1 (en) 2002-03-26 2006-08-25 RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)
PCT/US2006/062252 WO2007076328A2 (en) 2005-12-19 2006-12-18 Rna interference mediated inhibition of hepatitis c virus

Publications (1)

Publication Number Publication Date
NO20083208L true NO20083208L (no) 2008-09-18

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
NO20083208A NO20083208L (no) 2005-12-19 2008-07-18 RNA-interferensmediert inhibering av hepatitt C-virus-genekspresjon ved anvendelse av korte interfererende nukleinsyrer (siNA)

Country Status (7)

Country Link
US (1) US20080207542A1 (no)
EP (1) EP1987145A2 (no)
KR (1) KR20080079329A (no)
AU (1) AU2006330660A1 (no)
CA (1) CA2633684A1 (no)
NO (1) NO20083208L (no)
WO (1) WO2007076328A2 (no)

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US7935812B2 (en) 2002-02-20 2011-05-03 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA)
CA2581651C (en) 2004-10-01 2014-12-16 Novartis Vaccines And Diagnostics, Inc. Cholesterol-labelled modified rna
US8765704B1 (en) 2008-02-28 2014-07-01 Novartis Ag Modified small interfering RNA molecules and methods of use
US7404969B2 (en) * 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
AU2006336384B2 (en) 2005-02-14 2010-12-16 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
US20110125075A1 (en) * 2005-07-15 2011-05-26 National University Corporation Nagoya University Method for control of electroporation apparatus
WO2009029681A2 (en) 2007-08-27 2009-03-05 The Regents Of The University Of California Micrornas for inhibiting viral replication
US20110111044A1 (en) * 2008-07-31 2011-05-12 Enzon Pharmaceuticals, Inc. Nanoparticle compositions for nucleic acids delivery system
WO2010061881A1 (ja) * 2008-11-26 2010-06-03 中外製薬株式会社 Ｃ型肝炎ウイルスの働きを阻害するオリゴリボヌクレオチドまたはペプチド核酸
WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
EA026374B1 (ru) 2009-12-23 2017-04-28 Новартис Аг Липиды, липидные композиции и способы их применения
SG188497A1 (en) 2010-09-22 2013-05-31 Alios Biopharma Inc Substituted nucleotide analogs
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
WO2013040429A1 (en) 2011-09-14 2013-03-21 Rana Therapeutics Inc. Multimeric oligonucleotide compounds
CA2860234A1 (en) 2011-12-22 2013-06-27 Alios Biopharma, Inc. Substituted phosphorothioate nucleotide analogs
US8916538B2 (en) 2012-03-21 2014-12-23 Vertex Pharmaceuticals Incorporated Solid forms of a thiophosphoramidate nucleotide prodrug
WO2013142157A1 (en) 2012-03-22 2013-09-26 Alios Biopharma, Inc. Pharmaceutical combinations comprising a thionucleotide analog
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
JP2015523854A (ja) 2012-05-16 2015-08-20 ラナ セラピューティクス インコーポレイテッド Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法
CA2884608A1 (en) 2012-09-14 2014-03-20 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
US10342761B2 (en) 2014-07-16 2019-07-09 Novartis Ag Method of encapsulating a nucleic acid in a lipid nanoparticle host
CA2966044A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
EP3271460A4 (en) 2015-03-17 2019-03-13 The General Hospital Corporation INTERACTOME RNA OF COMPLEX REPRESSIVE POLYCOMB 1 (PRC1)
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US20030077829A1 (en) * 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
EP1670915A2 (en) * 2003-09-16 2006-06-21 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS (HCV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
EP1713915B1 (en) * 2004-02-10 2009-12-16 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING MULTIFUNCTIONAL SHORT INTERFERING NUCLEIC ACID (MULTIFUNCTIONAL siNA)

Also Published As

Publication number Publication date
KR20080079329A (ko) 2008-08-29
CA2633684A1 (en) 2007-07-05
US20080207542A1 (en) 2008-08-28
WO2007076328A2 (en) 2007-07-05
AU2006330660A1 (en) 2007-07-05
WO2007076328A3 (en) 2008-08-14
EP1987145A2 (en) 2008-11-05

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