US 11,866,701 B2
Complement component C3 iRNA compositions and methods of use thereof
Gregory Hinkle, Cambridge, MA (US); and Anna Borodovsky, Cambridge, MA (US)
Assigned to Alnylam Pharmaceuticals, Inc., Cambridge, MA (US)
Appl. No. 16/760,593
Filed by Alnylam Pharmaceuticals, Inc., Cambridge, MA (US)
PCT Filed Nov. 1, 2018, PCT No. PCT/US2018/058705
§ 371(c)(1), (2) Date Apr. 30, 2020,
PCT Pub. No. WO2019/089922, PCT Pub. Date May 9, 2019.
Claims priority of provisional application 62/580,030, filed on Nov. 1, 2017.
Prior Publication US 2021/0261959 A1, Aug. 26, 2021
Int. Cl. C12N 15/113 (2010.01); A61K 31/7115 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 31/7115 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/3235 (2013.01); C12N 2310/351 (2013.01)] 24 Claims
 
1. A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of complement component C3 in a cell, wherein said dsRNA comprises a sense strand and an antisense strand forming a double stranded region,
wherein each strand is independently 15-30 nucleotides in length,
wherein the antisense strand comprises at least 15 contiguous nucleotides from the complement of nucleotides 4614-4632 of SEQ ID NO:1,
wherein all of the nucleotides of the sense strand and all of the nucleotides of the antisense strand comprise a nucleotide modification, and
wherein at least one strand is conjugated to a ligand.