US 12,258,565 B2
Complement component C3 iRNA compositions and methods of use thereof
Mark Keating, Weston, MA (US); James D. McIninch, Burlington, MA (US); Elane Fishilevich, Rochester, MA (US); Kristina Yucius, Burlington, MA (US); Sarah Solomon, Cambridge, MA (US); Mark K. Schlegel, Boston, MA (US); Adam Castoreno, Framingham, MA (US); and Charalambos Kaittanis, Cambridge, MA (US)
Assigned to Alnylam Pharmaceuticals, Inc., Cambridge, MA (US)
Filed by Alnylam Pharmaceuticals, Inc., Cambridge, MA (US)
Filed on Mar. 4, 2024, as Appl. No. 18/594,132.
Application 18/594,132 is a division of application No. 17/721,530, filed on Apr. 15, 2022.
Application 17/721,530 is a continuation of application No. PCT/US2020/056563, filed on Oct. 21, 2020.
Claims priority of provisional application 62/924,210, filed on Oct. 22, 2019.
Prior Publication US 2024/0209369 A1, Jun. 27, 2024
Int. Cl. C07H 21/04 (2006.01); C12N 15/113 (2010.01); C12N 15/66 (2006.01)
CPC C12N 15/113 (2013.01) [C12N 15/66 (2013.01); C12N 2310/3125 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01)] 28 Claims
 
1. A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of complement component C3 in a cell, or a pharmaceutically acceptable salt thereof, comprising a sense strand differing by no more than 4 bases from the nucleotide sequence 5′-gsasgccgUfuCfUfCfuacaauuacu-3′ of SEQ ID NO:4188 and an antisense strand differing by no more than 4 bases from the nucleotide sequence 5′-asGfsuaaUfuGfUfagagAfaCfggcucsgsg-3′ of SEQ ID NO:4367,
wherein a, g, c and u are 2′-O-methyl (2′-OMe) A, G, C, and U, respectively; Af, Gf, Cf, and Uf are 2′-fluoro A, G, C and U, respectively; and s is a phosphorothioate linkage.