Title:
COMPOSITION FOR INHIBITING THE EXPRESSION OF TARGET GENE
Document Type and Number:
WIPO Patent Application WO/2011/007795
Kind Code:
A1
Abstract:
Disclosed are: a composition comprising a double-stranded nucleic acid molecule and a liposome in which the double-stranded nucleic acid molecule is encapsulated; and others. In the composition, the double-stranded nucleic acid molecule comprises an antisense strand and a sense strand, wherein the antisense strand is a polynucleotide which contains a nucleotide sequence complementary to a sequence composed of contiguous 17 nucleotides contained in mRNA for a target gene and which has a length of 17 to 30 nucleotides, the sense strand is a polynucleotide which contains a nucleotide sequence complementary to a sequence lying between the 1st nucleotide and the 17th nucleotide numbered from the 5'-terminal toward the 3'-terminal in the antisense strand and which has a length of 17 to 30 nucleotides, each of sugars respectively bound to a nucleotide located at a specific position in the antisense strand and a nucleotide located at a specific position in the sense strand is a deoxyribose or a ribose in which a hydroxy group at position-2' is substituted by a modifying group, and the deoxyribose and the ribose are contained at a specific ratio. In the composition, the liposome has a lipid bilayer membrane comprising a lipid derivative, fatty acid derivative or aliphatic hydrocarbon derivative of a water-soluble substance as a component.
Inventors:
SHINOHARA FUMIKAZU
YOSHIDA TETSUO
SUGISHITA HIROKO
NAOI TOMOYUKI
ISHII TOSHIHIKO
ENOKIZONO JUNICHI
YOSHIDA TETSUO
SUGISHITA HIROKO
NAOI TOMOYUKI
ISHII TOSHIHIKO
ENOKIZONO JUNICHI
Application Number:
PCT/JP2010/061884
Publication Date:
January 20, 2011
Filing Date:
July 14, 2010
Export Citation:
Assignee:
KYOWA HAKKO KIRIN CO LTD (JP)
SHINOHARA FUMIKAZU
YOSHIDA TETSUO
SUGISHITA HIROKO
NAOI TOMOYUKI
ISHII TOSHIHIKO
ENOKIZONO JUNICHI
SHINOHARA FUMIKAZU
YOSHIDA TETSUO
SUGISHITA HIROKO
NAOI TOMOYUKI
ISHII TOSHIHIKO
ENOKIZONO JUNICHI
International Classes:
A61K31/713; A61K9/127; A61K47/16; A61K47/18; A61K47/24; A61K47/34; A61P29/00; A61P35/00; C12N15/09
Domestic Patent References:
| WO2006080118A1 | 2006-08-03 | |||
| WO2010013815A1 | 2010-02-04 |
Foreign References:
| JP2009513151A | 2009-04-02 |
Other References:
JUDGE, A.D. ET AL.: "Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics in mice", J CLIN INVEST, vol. 119, no. 3, March 2009 (2009-03-01), pages 661 - 73, XP002618489, DOI: doi:10.1172/JCI37515
ALLERSON, C.R. ET AL.: "Fully 2'-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA", J MED CHEM, vol. 48, no. 4, 2005, pages 901 - 4, XP002404763, DOI: doi:10.1021/jm049167j
LAYZER, J.M. ET AL.: "In vivo activity of nuclease-resistant siRNAs", RNA, vol. 10, no. 5, 2004, pages 766 - 71, XP002598150, DOI: doi:10.1261/RNA.5239604
CHIU, Y.L. ET AL.: "siRNA function in RNAi: a chemical modification analysis", RNA, vol. 9, no. 9, 2003, pages 1034 - 48, XP008158876, DOI: doi:10.1261/rna.5103703
ALLERSON, C.R. ET AL.: "Fully 2'-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA", J MED CHEM, vol. 48, no. 4, 2005, pages 901 - 4, XP002404763, DOI: doi:10.1021/jm049167j
LAYZER, J.M. ET AL.: "In vivo activity of nuclease-resistant siRNAs", RNA, vol. 10, no. 5, 2004, pages 766 - 71, XP002598150, DOI: doi:10.1261/RNA.5239604
CHIU, Y.L. ET AL.: "siRNA function in RNAi: a chemical modification analysis", RNA, vol. 9, no. 9, 2003, pages 1034 - 48, XP008158876, DOI: doi:10.1261/rna.5103703
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