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Title:
COMPLEMENT COMPONENT IRNA COMPOSITIONS AND METHODS OF USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2015/089368
Kind Code:
A3
Abstract:
The invention relates to iRNA, e.g., double- stranded ribonucleic acid (dsRNA), compositions targeting the complement factor B (CFB) gene, the complement component C3 gene, and the complement component C9 gene and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of CFB, C9 and/or C3 and to treat subjects having a complement component-associated disease, e.g., paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.

Inventors:
BORODOVSKY, Anna (300 Third Street, 3rd FloorCambridge, MA, 02142, US)
BETTENCOURT, Brian (300 Third Street, 3rd FloorCambridge, MA, 02142, US)
Application Number:
US2014/069951
Publication Date:
August 20, 2015
Filing Date:
December 12, 2014
Export Citation:
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Assignee:
ALNYLAM PHARMACEUTICALS, INC. (300 Third Street, 3rd FloorCambridge, MA, 02142, US)
International Classes:
C12N15/113; A61K31/712; A61P25/28; A61P37/06; A61P43/00; C12N15/11; A61K31/713
Domestic Patent References:
WO2008036841A22008-03-27
WO2013074974A22013-05-23
WO2006047673A22006-05-04
WO2013067076A22013-05-10
Foreign References:
US20070088154A12007-04-19
EP1752536A12007-02-14
Other References:
NALINI S BORA ET AL: "Complement Activation via Alternative Pathway Is Critical in the Development of Laser-Induced Choroidal Neovascularization: Role of Factor B and Factor H", THE JOURNAL OF IMMUNOLOGY, THE AMERICAN ASSOCIATION OF IMMUNOLOGISTS, US, vol. 177, 1 January 2006 (2006-01-01), pages 1872 - 1878, XP007908456, ISSN: 0022-1767
ANNA BORODOVSKY ET AL: "Development Of RNAi Therapeutics Targeting The Complement Pathway", BLOOD, vol. 122, no. 21, 15 November 2013 (2013-11-15) - 10 December 2013 (2013-12-10), US, pages 2471, XP055126934, ISSN: 0006-4971
CHENG LING-LI ET AL: "[Effect of C5-siRNA silencing receptor C5 on myocardial ischemia injury in rats]", JOURNAL OF SOUTHERN MEDICAL UNIVERSITY - NAN FANG YI KE DA XUEBAO, NANFANG-YIKE-DAXUE , CN, vol. 30, no. 6, 1 June 2010 (2010-06-01), pages 1486 - 1488, XP008170341, ISSN: 1673-4254
Attorney, Agent or Firm:
ZACHARAKIS, Maria, Laccotripe et al. (McCarter & English, LLP265 Franklin Stree, Boston MA, 02110, US)
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