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WO2006128141A3 - Rna interference mediated inhibition of stromal cell-derived factor-1 (sdf-1) gene expression using short interfering nucleic acid (sina) - Google Patents

Rna interference mediated inhibition of stromal cell-derived factor-1 (sdf-1) gene expression using short interfering nucleic acid (sina) Download PDF

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Publication number
WO2006128141A3
WO2006128141A3 PCT/US2006/020846 US2006020846W WO2006128141A3 WO 2006128141 A3 WO2006128141 A3 WO 2006128141A3 US 2006020846 W US2006020846 W US 2006020846W WO 2006128141 A3 WO2006128141 A3 WO 2006128141A3
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WO
WIPO (PCT)
Prior art keywords
nucleic acid
acid molecules
sdf
rna
gene expression
Prior art date
Application number
PCT/US2006/020846
Other languages
French (fr)
Other versions
WO2006128141A2 (en
Inventor
Roberto Guerciolini
James Mcswiggen
Vasant Jadhav
Chandra Vargeese
Original Assignee
Sirna Therapeutics Inc
Roberto Guerciolini
James Mcswiggen
Vasant Jadhav
Chandra Vargeese
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 US11/140,328 external-priority patent/US20060019917A1/en
Priority claimed from US11/205,646 external-priority patent/US20080161256A1/en
Priority claimed from US11/234,730 external-priority patent/US20070270579A1/en
Priority claimed from US11/299,254 external-priority patent/US20060217331A1/en
Priority claimed from US11/353,630 external-priority patent/US7514099B2/en
Priority claimed from US11/369,108 external-priority patent/US20070160980A1/en
Application filed by Sirna Therapeutics Inc, Roberto Guerciolini, James Mcswiggen, Vasant Jadhav, Chandra Vargeese filed Critical Sirna Therapeutics Inc
Priority to EP06784507A priority Critical patent/EP1891217A2/en
Publication of WO2006128141A2 publication Critical patent/WO2006128141A2/en
Publication of WO2006128141A3 publication Critical patent/WO2006128141A3/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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • C12N2310/31—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

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

Abstract

The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of stromal cell-derived factor- 1 (SDF-I) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in stromal cell-derived factor- 1 (SDF-I) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including 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 capable of mediating RNA interference (RNAi) against stromal cell- derived factor- 1 (SDF-I) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate SDF-I gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce angiogenesis related diseases traits and conditions, including but not limited to ocular disease, cancer and proliferative diseases, traits, and conditions, and/or other disease states, conditions, or traits associated with SDF-I gene expression or activity in a subject or organism.
PCT/US2006/020846 2005-05-27 2006-05-26 Rna interference mediated inhibition of stromal cell-derived factor-1 (sdf-1) gene expression using short interfering nucleic acid (sina) WO2006128141A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP06784507A EP1891217A2 (en) 2005-05-27 2006-05-26 Rna interference mediated inhibition of stromal cell-derived factor-1 (sdf-1) gene expression using short interfering nucleic acid (sina)

Applications Claiming Priority (16)

Application Number Priority Date Filing Date Title
US11/140,328 US20060019917A1 (en) 2001-05-18 2005-05-27 RNA interference mediated inhibition of stromal cell-derived factor-1 (SDF-1) gene expression using short interfering nucleic acid (siNA)
US11/140,328 2005-05-27
US70394605P 2005-07-29 2005-07-29
US60/703,946 2005-07-29
US11/205,646 US20080161256A1 (en) 2001-05-18 2005-08-17 RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US11/205,646 2005-08-17
US11/234,730 US20070270579A1 (en) 2001-05-18 2005-09-23 RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US11/234,730 2005-09-23
US73702405P 2005-11-15 2005-11-15
US60/737,024 2005-11-15
US11/299,254 2005-12-08
US11/299,254 US20060217331A1 (en) 2001-05-18 2005-12-08 Chemically modified double stranded nucleic acid molecules that mediate RNA interference
US11/353,630 2006-02-14
US11/353,630 US7514099B2 (en) 2005-02-14 2006-02-14 Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
US11/369,108 2006-03-06
US11/369,108 US20070160980A1 (en) 2001-05-18 2006-03-06 RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)

Publications (2)

Publication Number Publication Date
WO2006128141A2 WO2006128141A2 (en) 2006-11-30
WO2006128141A3 true WO2006128141A3 (en) 2007-07-12

Family

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Family Applications (1)

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PCT/US2006/020846 WO2006128141A2 (en) 2005-05-27 2006-05-26 Rna interference mediated inhibition of stromal cell-derived factor-1 (sdf-1) gene expression using short interfering nucleic acid (sina)

Country Status (2)

Country Link
EP (1) EP1891217A2 (en)
WO (1) WO2006128141A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12297431B2 (en) 2009-09-11 2025-05-13 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression

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Publication number Priority date Publication date Assignee Title
US7514099B2 (en) 2005-02-14 2009-04-07 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
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)
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
PL3210633T3 (en) 2006-01-26 2019-12-31 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
WO2009102427A2 (en) 2008-02-11 2009-08-20 Rxi Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
EP2340310B1 (en) 2008-09-22 2015-06-03 RXi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
US11029313B2 (en) 2008-09-26 2021-06-08 The General Hospital Corporation Method of treating cervical neoplasia in patients infected with human papilloma virus
US9229004B2 (en) 2008-09-26 2016-01-05 The General Hospital Corporation Methods for detecting and treating cancer
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
US9340786B2 (en) 2010-03-24 2016-05-17 Rxi Pharmaceuticals Corporation RNA interference in dermal and fibrotic indications
EP2550001B1 (en) 2010-03-24 2019-05-22 Phio Pharmaceuticals Corp. Rna interference in ocular indications
RU2615143C2 (en) 2010-03-24 2017-04-04 Адвирна Self-delivered rnai compounds of reduced size
CN106061488B (en) 2013-12-02 2021-04-09 菲奥医药公司 Immunotherapy for Cancer
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
CN107073294A (en) 2014-09-05 2017-08-18 阿克赛医药公司 Use the method for targeting TYR or MMP1 exonuclease treatment aging and skin disorder
CA2991598A1 (en) 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
CA3002744A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12297431B2 (en) 2009-09-11 2025-05-13 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression

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EP1891217A2 (en) 2008-02-27

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