WO2007120527A3 - Rna interference mediated inhibition of respiratory syncytial virus (rsv) expression using short interfering nucleic acid (sina) - Google Patents

Rna interference mediated inhibition of respiratory syncytial virus (rsv) expression using short interfering nucleic acid (sina) Download PDF

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Publication number
WO2007120527A3
WO2007120527A3 PCT/US2007/008186 US2007008186W WO2007120527A3 WO 2007120527 A3 WO2007120527 A3 WO 2007120527A3 US 2007008186 W US2007008186 W US 2007008186W WO 2007120527 A3 WO2007120527 A3 WO 2007120527A3
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WO
WIPO (PCT)
Prior art keywords
nucleic acid
rsv
expression
short interfering
sina
Prior art date
Application number
PCT/US2007/008186
Other languages
French (fr)
Other versions
WO2007120527A2 (en
Inventor
Narendra K Vaish
Chandra Vargeese
James Mcswiggen
Original Assignee
Sirna Therapeutics Inc
Narendra K Vaish
Chandra Vargeese
James Mcswiggen
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 US11/395,833 external-priority patent/US20060287267A1/en
Application filed by Sirna Therapeutics Inc, Narendra K Vaish, Chandra Vargeese, James Mcswiggen filed Critical Sirna Therapeutics Inc
Publication of WO2007120527A2 publication Critical patent/WO2007120527A2/en
Publication of WO2007120527A3 publication Critical patent/WO2007120527A3/en

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    • 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
    • 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 N.A.
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/321—2'-O-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
    • C12N2310/32—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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

This invention relates to compounds, compositions, and methods useful for modulating sespiratory syncytial virus (RSV) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of RSV gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of RSV genes, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such such small nucleic acid molecules cocktails thereof. The application also relates to methods of treating diseases and conditions associated with RSV gene expression, such as RSV infection, respiratory failure, bronchiolitis and pneumonia, as well as providing dosing regimens and treatment protocols.
PCT/US2007/008186 2006-03-31 2007-03-30 Rna interference mediated inhibition of respiratory syncytial virus (rsv) expression using short interfering nucleic acid (sina) WO2007120527A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11/395,833 2006-03-31
US11/395,833 US20060287267A1 (en) 2001-05-18 2006-03-31 RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)

Publications (2)

Publication Number Publication Date
WO2007120527A2 WO2007120527A2 (en) 2007-10-25
WO2007120527A3 true WO2007120527A3 (en) 2008-07-03

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2007/008186 WO2007120527A2 (en) 2006-03-31 2007-03-30 Rna interference mediated inhibition of respiratory syncytial virus (rsv) expression using short interfering nucleic acid (sina)

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WO (1) WO2007120527A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2005056021A1 (en) * 2003-12-04 2005-06-23 University Of South Florida Polynucleotides for reducing respiratory syncytial virus gene expression
US20060287267A1 (en) * 2001-05-18 2006-12-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060287267A1 (en) * 2001-05-18 2006-12-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2005056021A1 (en) * 2003-12-04 2005-06-23 University Of South Florida Polynucleotides for reducing respiratory syncytial virus gene expression

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
BARIK SAILEN: "Control of nonsegmented negative-strand RNA virus replication by siRNA", VIRUS RESEARCH, AMSTERDAM, NL, vol. 102, no. 1, June 2004 (2004-06-01), pages 27 - 35, XP002410942, ISSN: 0168-1702 *
BITKO VIRA ET AL: "Inhibition of respiratory viruses by nasally administered siRNA", NATURE MEDICINE, NATURE PUBLISHING GROUP, NEW YORK, NY, US, vol. 11, no. 1, January 2005 (2005-01-01), pages 50 - 55, XP002410945, ISSN: 1078-8956 *
CHOUDHARY SANJEEV ET AL: "Respiratory syncytial virus influences NF-kappa B-dependent gene expression through a novel pathway involving MAP3K14/NIK expression and nuclear complex formation with NF-kappa B2", JOURNAL OF VIROLOGY, vol. 79, no. 14, July 2005 (2005-07-01), pages 8948 - 8959, XP002467145, ISSN: 0022-538X *
DORSETT Y ET AL: "SIRNAS: APPLICATIONS IN FUNCTIONAL GENOMICS AND POTENTIAL AS THERAPEUTICS", NATURE, NATURE PUBLISHING GROUP, LONDON, GB, vol. 3, no. 4, April 2004 (2004-04-01), pages 318 - 329, XP009042544, ISSN: 0028-0836 *
LEONARD J N ET AL: "Antiviral RNAi therapy: emerging approaches for hitting a moving target.", GENE THERAPY MAR 2006, vol. 13, no. 6, 22 September 2005 (2005-09-22), pages 532 - 540, XP002467144, ISSN: 0969-7128 *
MANOHARAN M: "RNA interference and chemically modified small interfering RNAs", CURRENT OPINION IN CHEMICAL BIOLOGY, CURRENT BIOLOGY LTD, LONDON, GB, vol. 8, no. 6, December 2004 (2004-12-01), pages 570 - 579, XP004649493, ISSN: 1367-5931 *
XU ZAN ET AL: "Potent inhibition of respiratory syncytial virus by combination treatment with 2-5A antisense and ribavirin", ANTIVIRAL RESEARCH, ELSEVIER SCIENCE BV., AMSTERDAM, NL, vol. 61, no. 3, March 2004 (2004-03-01), pages 195 - 206, XP002410944, ISSN: 0166-3542 *

Also Published As

Publication number Publication date
WO2007120527A2 (en) 2007-10-25

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