(19)
(11)EP 3 312 281 A3

(12)EUROPEAN PATENT APPLICATION

(88)Date of publication A3:
27.06.2018 Bulletin 2018/26

(43)Date of publication A2:
25.04.2018 Bulletin 2018/17

(21)Application number: 17187047.0

(22)Date of filing:  13.03.2014
(51)International Patent Classification (IPC): 
C12N 15/113(2010.01)
A61K 31/7088(2006.01)
(84)Designated Contracting States:
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
Designated Extension States:
BA ME

(30)Priority: 14.03.2013 US 201361782531 P
20.06.2013 US 201361837399 P
15.11.2013 US 201361904579 P
06.12.2013 US 201361912777 P
20.02.2014 US 201461942367 P

(62)Application number of the earlier application in accordance with Art. 76 EPC:
14722018.0 / 2970974

(71)Applicant: Alnylam Pharmaceuticals, Inc.
Cambridge, MA 02142 (US)

(72)Inventors:
  • FITZGERALD, Kevin
    Cambridge, MA 02142 (US)
  • BUTLER, James
    Cambridge, MA 02142 (US)
  • BETTENCOURT, Brian
    Cambridge, MA 02142 (US)
  • BORODOVSKY, Anna
    Cambridge, MA 02142 (US)
  • KUCHIMANCHI, Satyanarayana
    Cambridge, MA 02142 (US)
  • CHARISSE, Klaus
    Cambridge, MA 02142 (US)
  • MANOHARAN, Muthiah
    Cambridge, MA 02142 (US)
  • MAIER, Martin
    Cambridge, MA 02142 (US)
  • RAJEEV, Kallanthottahil G.
    Cambridge, MA 02142 (US)
  • FOSTER, Donald
    Cambridge, MA 02142 (US)

(74)Representative: Hoffmann Eitle 
Patent- und Rechtsanwälte PartmbB Arabellastraße 30
81925 München
81925 München (DE)

  


(54)COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF


(57) The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.







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