Title:
COMPOSITIONS AND METHODS FOR INHIBITION OF EXPRESSION OF PROTEIN C (PROC) GENES
Document Type and Number:
WIPO Patent Application WO/2012/177949
Kind Code:
A3
Abstract:
The invention relates to double-stranded ribonucleic acid (dsRNA) targeting a PROC gene, and methods of using the dsRNA to inhibit expression of PROC. At least one nucleotide ofthe dsRNA can be a modified nucleotide, e.g., a 2 -0-methyl modified nucleotide, a nucleotide comprising a 5'-phosphorothioate group, and a terminal nucleotide linked to a cholesteryl derivative or dodecanoic acid bisdecylamide group. Other examples of modified nucleotides include a 2'-deoxy-2'-fluoro modified nucleotide, a 2'-deoxymodified nucleotide, a locked nucleotide, an abasic nucleotide, 2'-amino-modified nucleotide, 2' -alkyl-modified nucleotide, morpholino nucleotide, a phosphoramidate, and a non-natural base comprising nucleotide. A dsRNA of the invention can include one or more of any of these modified nucleotides.
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Inventors:
TOUDJARSKA IVANKA (US)
MARAGANORE JOHN M (US)
BETTENCOURT BRIAN (US)
MILSTEIN STUART (US)
MAIER MARTIN (US)
CHARISSE KLAUS (US)
KALLANTHOTTATHIL RAJEEV (US)
KUCHIMANCHI SATYANARAYANA (US)
MARAGANORE JOHN M (US)
BETTENCOURT BRIAN (US)
MILSTEIN STUART (US)
MAIER MARTIN (US)
CHARISSE KLAUS (US)
KALLANTHOTTATHIL RAJEEV (US)
KUCHIMANCHI SATYANARAYANA (US)
Application Number:
PCT/US2012/043644
Publication Date:
May 30, 2013
Filing Date:
June 21, 2012
Export Citation:
Assignee:
ALNYLAM PHARMACEUTICALS INC (US)
TOUDJARSKA IVANKA (US)
MARAGANORE JOHN M (US)
BETTENCOURT BRIAN (US)
MILSTEIN STUART (US)
MAIER MARTIN (US)
CHARISSE KLAUS (US)
KALLANTHOTTATHIL RAJEEV (US)
KUCHIMANCHI SATYANARAYANA (US)
TOUDJARSKA IVANKA (US)
MARAGANORE JOHN M (US)
BETTENCOURT BRIAN (US)
MILSTEIN STUART (US)
MAIER MARTIN (US)
CHARISSE KLAUS (US)
KALLANTHOTTATHIL RAJEEV (US)
KUCHIMANCHI SATYANARAYANA (US)
International Classes:
A61K31/713
Domestic Patent References:
| WO2011030332A2 | 2011-03-17 |
Foreign References:
| US20100184822A1 | 2010-07-22 | |||
| US20100184672A1 | 2010-07-22 | |||
| US20050019927A1 | 2005-01-27 |
Other References:
"Protein C siRNA (h):sc-72054", SANTA CRUZ BIOTECHNOLOGY, 27 March 2009 (2009-03-27), XP055069592, Retrieved from the Internet [retrieved on 20130106]
SEHGEL ET AL.: "RNAi-Mediated Inhibition of Natural Anticoagulants for Treatment of Hemophilia", ALNYLAN PHARMACEUTICALS, 3 July 2012 (2012-07-03), XP055069594, Retrieved from the Internet [retrieved on 20130106]
TOUDJARSKA ET AL.: "RNAi-Mediated Inhibition of Activated Protein C - A New Approach for Hemophilia Treatment", ALNYLAN PHARMACEUTICALS, 12 December 2011 (2011-12-12), XP055069607, Retrieved from the Internet [retrieved on 20130106]
"PROC (ID 5624) Trilencer-27 Human siRNA", 2012, XP008185894, Retrieved from the Internet [retrieved on 20130106]
SEHGEL ET AL.: "RNAi-Mediated Inhibition of Natural Anticoagulants for Treatment of Hemophilia", ALNYLAN PHARMACEUTICALS, 3 July 2012 (2012-07-03), XP055069594, Retrieved from the Internet
TOUDJARSKA ET AL.: "RNAi-Mediated Inhibition of Activated Protein C - A New Approach for Hemophilia Treatment", ALNYLAN PHARMACEUTICALS, 12 December 2011 (2011-12-12), XP055069607, Retrieved from the Internet
"PROC (ID 5624) Trilencer-27 Human siRNA", 2012, XP008185894, Retrieved from the Internet
Attorney, Agent or Firm:
SHUSTER, Michael, J. et al. (Silicon Valley Center801 California Stree, Mountain View CA, US)
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