CA2884658A1 - Modified small interfering rna molecules and methods of use - Google Patents

Modified small interfering rna molecules and methods of use Download PDF

Info

Publication number
CA2884658A1
CA2884658A1 CA 2884658 CA2884658A CA2884658A1 CA 2884658 A1 CA2884658 A1 CA 2884658A1 CA 2884658 CA2884658 CA 2884658 CA 2884658 A CA2884658 A CA 2884658A CA 2884658 A1 CA2884658 A1 CA 2884658A1
Authority
CA
Canada
Prior art keywords
sirna
sequence
strand
virus
modified
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
CA 2884658
Other languages
English (en)
French (fr)
Inventor
Jang Han
Mi Young Seo
Michael Houghton
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Novartis Vaccines and Diagnostics Inc
Original Assignee
Novartis Vaccines and Diagnostics 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
Application filed by Novartis Vaccines and Diagnostics Inc filed Critical Novartis Vaccines and Diagnostics Inc
Publication of CA2884658A1 publication Critical patent/CA2884658A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Pulmonology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Processes Of Treating Macromolecular Substances (AREA)
CA 2884658 2002-07-26 2003-07-25 Modified small interfering rna molecules and methods of use Abandoned CA2884658A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US39860502P 2002-07-26 2002-07-26
US60/398,605 2002-07-26
US46183803P 2003-04-11 2003-04-11
US60/461,838 2003-04-11
US47023003P 2003-05-14 2003-05-14
US60/470,230 2003-05-14
CA2493949A CA2493949C (en) 2002-07-26 2003-07-25 Modified small interfering rna molecules and methods of use

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CA2493949A Division CA2493949C (en) 2002-07-26 2003-07-25 Modified small interfering rna molecules and methods of use

Publications (1)

Publication Number Publication Date
CA2884658A1 true CA2884658A1 (en) 2004-02-05

Family

ID=32397947

Family Applications (2)

Application Number Title Priority Date Filing Date
CA 2884658 Abandoned CA2884658A1 (en) 2002-07-26 2003-07-25 Modified small interfering rna molecules and methods of use
CA2493949A Expired - Fee Related CA2493949C (en) 2002-07-26 2003-07-25 Modified small interfering rna molecules and methods of use

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2493949A Expired - Fee Related CA2493949C (en) 2002-07-26 2003-07-25 Modified small interfering rna molecules and methods of use

Country Status (9)

Country Link
US (4) US20050058982A1 (https=)
EP (1) EP1532248B1 (https=)
JP (2) JP4709545B2 (https=)
AT (1) ATE427352T1 (https=)
AU (1) AU2003261231A1 (https=)
CA (2) CA2884658A1 (https=)
DE (1) DE60326961D1 (https=)
ES (1) ES2322145T3 (https=)
WO (1) WO2004011647A1 (https=)

Families Citing this family (88)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
ATE556714T1 (de) 2002-02-01 2012-05-15 Life Technologies Corp Doppelsträngige oligonukleotide
EP1572902B1 (en) * 2002-02-01 2014-06-11 Life Technologies Corporation HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
AU2003219817B2 (en) * 2002-02-20 2006-08-31 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hepatitis C virus
US20090192105A1 (en) * 2002-02-20 2009-07-30 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF INTERCELLULAR ADHESION MOLECULE (ICAM) GENE EXPRESSION USING SHORT INTERFERING NUCELIC ACID (siNA)
US20040248094A1 (en) * 2002-06-12 2004-12-09 Ford Lance P. Methods and compositions relating to labeled RNA molecules that reduce gene expression
GB2406169B (en) * 2002-06-12 2006-11-01 Ambion Inc Methods and compositions relating to labeled rna molecules that reduce gene expression
US20100075423A1 (en) * 2002-06-12 2010-03-25 Life Technologies Corporation Methods and compositions relating to polypeptides with rnase iii domains that mediate rna interference
AU2003261231A1 (en) 2002-07-26 2004-02-16 Chiron Corporation Modified small interfering rna molecules and methods of use
US20040029275A1 (en) * 2002-08-10 2004-02-12 David Brown Methods and compositions for reducing target gene expression using cocktails of siRNAs or constructs expressing siRNAs
US20060160759A1 (en) * 2002-09-28 2006-07-20 Jianzhu Chen Influenza therapeutic
DE10302421A1 (de) * 2003-01-21 2004-07-29 Ribopharma Ag Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit
WO2004092383A2 (en) * 2003-04-15 2004-10-28 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF SEVERE ACUTE RESPIRATORY SYNDROME (SARS) VIRUS GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
AU2004263830B2 (en) 2003-06-13 2008-12-18 Alnylam Pharmaceuticals, Inc. Double-stranded ribonucleic acid with increased effectiveness in an organism
US20050043266A1 (en) * 2003-07-25 2005-02-24 Sumedha Jayasena Short interfering RNA as an antiviral agent for hepatitis C
WO2005040388A2 (en) * 2003-08-22 2005-05-06 Nucleonics Inc. Eukariotic expression systems for expression of inhibitory rna in multiple intracellular compartments
US20050060771A1 (en) * 2003-09-11 2005-03-17 Farmer Andrew Alan siRNA encoding constructs and methods for using the same
US7371802B2 (en) * 2003-10-28 2008-05-13 Basell Poliolefine Italia S.R.L. Components and catalysts for the polymerization of olefins
CA2558771C (en) 2004-03-05 2013-01-08 Benitec, Inc. Multiple promoter expression cassettes for simultaneous delivery of rnai agents
CA2559955C (en) * 2004-03-15 2016-02-16 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20050244869A1 (en) * 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
WO2005111219A1 (en) * 2004-04-16 2005-11-24 University Of Washington METHODS AND VECTORS FOR EXPRESSING siRNA
US20060014289A1 (en) * 2004-04-20 2006-01-19 Nastech Pharmaceutical Company Inc. Methods and compositions for enhancing delivery of double-stranded RNA or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells
AU2005247509C1 (en) * 2004-05-27 2012-09-20 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
US7297786B2 (en) 2004-07-09 2007-11-20 University Of Iowa Research Foundation RNA interference in respiratory epitheial cells
US8361976B2 (en) 2004-07-09 2013-01-29 University Of Massachusetts Therapeutic alteration of transplantable tissues through in situ or ex vivo exposure to RNA interference molecules
EP1791567B1 (en) 2004-08-10 2015-07-29 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
DE602005027479D1 (de) 2004-09-24 2011-05-26 Alnylam Pharmaceuticals Inc Targeting von zwischenprodukten zur gegenstrangreplikation von einzelstrangigen viren durch rnai
US8765704B1 (en) 2008-02-28 2014-07-01 Novartis Ag Modified small interfering RNA molecules and methods of use
WO2006039656A2 (en) * 2004-10-01 2006-04-13 Novartis Vaccines And Diagnostics Inc. Modified small interfering rna molecules and methods of use
CA2587493A1 (en) * 2004-11-30 2006-06-08 Aeras Global Tb Vaccine Foundation Bacterial packaging strains usefuel for generation and production of recombinant double-stranded rna nucleocapsids and uses thereof
US20060142228A1 (en) * 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
NZ560936A (en) * 2005-02-03 2010-04-30 Benitec Inc RNAI expression constructs
CN1313159C (zh) * 2005-06-03 2007-05-02 陈志南 HAb18G/CD147分子小片段干涉RNA药物及其用途
WO2007053696A2 (en) * 2005-11-01 2007-05-10 Alnylam Pharmaceuticals, Inc. Rnai inhibition of influenza virus replication
EP2905336A1 (en) 2007-03-29 2015-08-12 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of a gene from the ebola
CN101686939B (zh) * 2007-04-17 2013-03-27 巴克斯特国际公司 用于肺部投送的核酸微粒
AR066984A1 (es) * 2007-06-15 2009-09-23 Novartis Ag Inhibicion de la expresion de la subunidad alfa del canal epitelial de sodio (enac) por medio de arni (arn de interferencia)
WO2009008645A2 (en) * 2007-07-06 2009-01-15 Mogam Biotechnology Research Institute Linear double-stranded rna molecule interfering with different target genes
EP2195428B1 (en) 2007-09-19 2013-12-11 Applied Biosystems, LLC SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
KR101070654B1 (ko) 2008-06-11 2011-10-07 건국대학교 산학협력단 중증급성호흡기증후군 코로나바이러스(ｓｃｖ)에 대한 저해활성을 가지는 조성물
JP2011190176A (ja) * 2008-07-07 2011-09-29 Kyowa Hakko Kirin Co Ltd 肥満細胞の脱顆粒抑制剤
RU2011132045A (ru) * 2008-12-31 2013-02-10 Эбботт Лэборетриз Праймеры и зонды для детектирования вируса гепатита с
WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
JPWO2010137570A1 (ja) * 2009-05-29 2012-11-15 学校法人慶應義塾 抗ウイルス剤または抗癌剤
US8435961B2 (en) * 2009-06-26 2013-05-07 Massachusetts Institute Of Technology Methods and compositions for increasing the activity of inhibitory RNA
US8268550B2 (en) * 2009-06-26 2012-09-18 Massachusetts Institute Of Technology Compositions and methods for identification of PARP function, inhibitors, and activators
WO2010151664A2 (en) 2009-06-26 2010-12-29 Massachusetts Institute Of Technology Compositions and methods for treating cancer and modulating stress granule formation
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
AP2015008874A0 (en) 2009-08-14 2015-11-30 Alnylam Pharmaceuticals Inc Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
PL2563920T3 (pl) 2010-04-29 2017-08-31 Ionis Pharmaceuticals, Inc. Modulacja ekspresji transtyretyny
US9029341B2 (en) * 2010-08-17 2015-05-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hepatitis B virus (HBV) gene expression using short interfering nucleic acid (siNA)
US8217163B2 (en) * 2010-09-20 2012-07-10 Biomics Biotechnologies Co., Ltd. Application of highly conserved domain sequences from viral genome as template to design therapeutic slirnas
EP2817287B1 (en) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
KR101520383B1 (ko) 2012-08-02 2015-05-15 에이비온 주식회사 Ｈｐｖ 감염과 관련된 암의 치료용 조성물
US9840703B2 (en) 2013-01-11 2017-12-12 Children's Medical Center Corporation Methods and compositions for the production of siRNAs
AU2014340149B2 (en) 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
US9616114B1 (en) 2014-09-18 2017-04-11 David Gordon Bermudes Modified bacteria having improved pharmacokinetics and tumor colonization enhancing antitumor activity
JP6767976B2 (ja) 2014-12-05 2020-10-14 トランスレイト バイオ， インコーポレイテッド 関節疾患の治療のためのメッセンジャーｒｎａ治療法
WO2016149508A1 (en) 2015-03-19 2016-09-22 Shire Human Genetic Therapies, Inc. Mrna therapy for pompe disease
US10676723B2 (en) 2015-05-11 2020-06-09 David Gordon Bermudes Chimeric protein toxins for expression by therapeutic bacteria
WO2016183366A2 (en) * 2015-05-12 2016-11-17 Protiva Biotherapeutics, Inc. Compositions and methods for silencing expression of hepatitis d virus rna
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
US11129906B1 (en) 2016-12-07 2021-09-28 David Gordon Bermudes Chimeric protein toxins for expression by therapeutic bacteria
US11180535B1 (en) 2016-12-07 2021-11-23 David Gordon Bermudes Saccharide binding, tumor penetration, and cytotoxic antitumor chimeric peptides from therapeutic bacteria
EP3556764A4 (en) 2016-12-16 2021-01-06 Gifu University NUCLEOSIDE DERIVATIVE AND USE FOR IT
TN2019000308A1 (en) 2017-07-06 2021-05-07 Arrowhead Pharmaceuticals Inc RNAi AGENTS FOR INHIBITING EXPRESSION OF ALPHA-ENaC AND METHODS OF USE
JP7173467B2 (ja) 2017-10-31 2022-11-16 ヤマサ醤油株式会社 ヌクレオシド誘導体及びその利用
US20230144739A1 (en) * 2020-03-16 2023-05-11 Medesis Pharma Treatment of covid-19 with reverse micelle system comprising unmodified oligonucleotides
EP4121018A4 (en) 2020-03-17 2024-07-03 Genevant Sciences Gmbh CATIONIC LIPIDS FOR LIPID DNANOPARTICLE DELIVERY OF THERAPEUTICS TO HEPATIC STELLA CELLS
CN115884796A (zh) * 2020-03-18 2023-03-31 南京强新生物医药有限公司 靶向冠状病毒基因的基因沉默剂及其用途
WO2021226479A1 (en) * 2020-05-07 2021-11-11 The Regents Of The University Of California Inhaled statins for treatment of viral respiratory diseases
AU2021403156A1 (en) 2020-12-18 2023-07-13 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles
KR20240146674A (ko) 2022-01-31 2024-10-08 제네반트 사이언시즈 게엠베하 지질 나노입자를 위한 이온화 가능한 양이온성 지질
AU2023255614A1 (en) 2022-04-18 2024-10-24 Vertex Pharmaceuticals Incorporated Compositions and methods for enhancing aav therapy and decreasing tropism of aav to the liver.
GB202208347D0 (en) 2022-06-07 2022-07-20 Univ Court Univ Of Glasgow Targets for cancer therapy
AU2023408479A1 (en) 2022-12-19 2025-05-01 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar)
EP4562158A1 (en) 2023-06-13 2025-06-04 Arnatar Therapeutics, Inc Advanced rna targeting (arnatar) for angiotensinogen
WO2025052278A1 (en) 2023-09-05 2025-03-13 Genevant Sciences Gmbh Pyrrolidine based cationic lipids for lipid nanoparticle delivery of therapeutics to hepatic stellate cells
WO2025133951A1 (en) 2023-12-21 2025-06-26 Genevant Sciences Gmbh Ionizable lipids suitable for lipid nanoparticles
WO2025165891A1 (en) 2024-01-29 2025-08-07 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof
EP4671372A3 (en) 2024-01-29 2026-03-11 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof
WO2026022656A2 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Lipid particle formulations with permanently charged cationic lipids
WO2026022677A1 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Permanently charged cationic lipids

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6190864B1 (en) * 1991-05-08 2001-02-20 Chiron Corporation HCV genomic sequences for diagnostics and therapeutics
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
US5610054A (en) * 1992-05-14 1997-03-11 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecule targeted against Hepatitis C virus
US20030206887A1 (en) * 1992-05-14 2003-11-06 David Morrissey RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA)
US6174868B1 (en) * 1992-09-10 2001-01-16 Isis Pharmaceuticals, Inc. Compositions and methods for treatment of hepatitis C virus-associated diseases
US5922857A (en) 1992-09-28 1999-07-13 Chiron Corporation Methods and compositions for controlling translation of HCV proteins
US5624803A (en) * 1993-10-14 1997-04-29 The Regents Of The University Of California In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides derived therefrom
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
WO1995030746A1 (en) * 1994-05-10 1995-11-16 The General Hospital Corporation Antisense inhibition of hepatitis c virus
US6107027A (en) * 1994-12-14 2000-08-22 University Of Washington Ribozymes for treating hepatitis C
US5874565A (en) * 1995-08-29 1999-02-23 Washington University Nucleic acids comprising a highly conserved novel 3 terminal sequence element of the hepatitis C virus
NZ507848A (en) 1996-10-28 2005-01-28 Univ Washington Method of increasing the mutation rate of a virus in a non-human by administering an RNA nucleoside analogue to a virally infected cell
US6133246A (en) * 1997-08-13 2000-10-17 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the modulation of JNK proteins
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US20020155582A1 (en) * 1999-12-23 2002-10-24 Lemon Stanley M. Replication competent hepatitis C virus and methods of use
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
KR20030005197A (ko) 2000-02-18 2003-01-17 샤이어 바이오켐 인코포레이티드 뉴클레오시드유도체를 이용한 플라비바이러스 감염의 치료또는 예방 방법
ES2336887T5 (es) * 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
MY164523A (en) 2000-05-23 2017-12-29 Univ Degli Studi Cagliari Methods and compositions for treating hepatitis c virus
US20030008841A1 (en) 2000-08-30 2003-01-09 Rene Devos Anti-HCV nucleoside derivatives
WO2002032920A2 (en) 2000-10-18 2002-04-25 Pharmasset Limited Modified nucleosides for treatment of viral infections and abnormal cellular proliferation
SK286630B6 (sk) * 2001-01-22 2009-02-05 Merck & Co., Inc. Nukleozidové deriváty, farmaceutický prostriedok s ich obsahom a ich použitie
EP1383782A1 (en) * 2001-03-26 2004-01-28 Sirna Therpeutics, Inc. Oligonucleotide mediated inhibition of hepatitis b virus and hepatitis c virus replication
EP1386004A4 (en) * 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
US20050209180A1 (en) * 2001-05-18 2005-09-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA)
GB0114286D0 (en) 2001-06-12 2001-08-01 Hoffmann La Roche Nucleoside Derivatives
DK2280070T3 (en) * 2001-07-23 2015-08-24 Univ Leland Stanford Junior Methods and compositions for use in RNAi-mediated inhibition of gene expression in mammals
US20030219823A1 (en) * 2001-09-07 2003-11-27 Alsobrook John P. Therapeutic polypeptides, nucleic acids encoding same, and methods of use
AU2003219817B2 (en) * 2002-02-20 2006-08-31 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hepatitis C virus
AU2003261231A1 (en) 2002-07-26 2004-02-16 Chiron Corporation Modified small interfering rna molecules and methods of use
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
DE10302421A1 (de) 2003-01-21 2004-07-29 Ribopharma Ag Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit
US20050043266A1 (en) * 2003-07-25 2005-02-24 Sumedha Jayasena Short interfering RNA as an antiviral agent for hepatitis C
WO2005116204A1 (ja) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
AR052918A1 (es) * 2005-02-25 2007-04-11 Recordati Ireland Ltd Clorhidrato de lercanidipina amorfo
AR066984A1 (es) * 2007-06-15 2009-09-23 Novartis Ag Inhibicion de la expresion de la subunidad alfa del canal epitelial de sodio (enac) por medio de arni (arn de interferencia)
EP3000885B1 (en) * 2009-12-18 2018-07-25 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat hsf1-related diseases

Also Published As

Publication number Publication date
AU2003261231A1 (en) 2004-02-16
EP1532248B1 (en) 2009-04-01
JP2006320327A (ja) 2006-11-30
EP1532248A1 (en) 2005-05-25
CA2493949C (en) 2015-06-02
WO2004011647A1 (en) 2004-02-05
US20050058982A1 (en) 2005-03-17
US20150361432A1 (en) 2015-12-17
CA2493949A1 (en) 2004-02-05
DE60326961D1 (de) 2009-05-14
AU2003261231A8 (en) 2004-02-16
JP4718379B2 (ja) 2011-07-06
WO2004011647A8 (en) 2004-06-10
ATE427352T1 (de) 2009-04-15
ES2322145T3 (es) 2009-06-17
JP2005533517A (ja) 2005-11-10
US20140073683A1 (en) 2014-03-13
US9139833B2 (en) 2015-09-22
US20120122962A1 (en) 2012-05-17
JP4709545B2 (ja) 2011-06-22

Similar Documents

Publication Publication Date Title
EP1532248B1 (en) Modified small interfering rna molecules and methods of use
CA2581651C (en) Cholesterol-labelled modified rna
CN108136206B (zh) 抗乙型肝炎病毒的组合物和药剂及其用途
CN103333890B (zh) 治疗乙型病毒性肝炎的rna干扰制剂
CN101970662A (zh) 带有修饰碱基的寡核苷酸作为抗病毒剂的应用
WO2021130266A1 (en) Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
US9084808B2 (en) Modified small interfering RNA molecules and methods of use
US20060128617A1 (en) Oligoribonucleotide or peptidic nucleic acid inhibiting function of hepatitis c virus
JP2023506540A (ja) Ｂ型肝炎ウイルス感染を処置するためのｓｃａｍｐ３阻害剤の使用
JP2025094072A (ja) Ｂ型肝炎ウイルス感染を処置するためのｃｏｐｓ３阻害剤の使用
JP2023506954A (ja) Ｂ型肝炎ウイルス感染を処置するためのｓａｒａｆ阻害剤の使用
WO2007084359A2 (en) Compositions and methods for the treatment of influenza infection
JP2023506550A (ja) Ｂ型肝炎ウイルス感染症を処置するためのｓｂｄｓ阻害剤の使用
Ely et al. 799. Expressed Long Hairpin RNA Sequences Inhibit HBV Replication In Vivo without Inducing an Interferon Response

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20150310

FZDE Discontinued

Effective date: 20180725