Patent ID: 11965166
Assignee: ALNYLAM PHARMACEUTICALS, INC.
Field: Biotechnology (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 25:
26. A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of complement factor B (CFB) in a cell, or a pharmaceutically acceptable salt thereof,
wherein the dsRNA, or a pharmaceutically acceptable salt thereof, comprises a sense strand and an antisense strand forming a double stranded region,
wherein the sense strand consists of the nucleotide sequence 5′-gsasauuccuGfAfAfuuuuaugacu-3′ of SEQ ID NO:1271 and the antisense strand consists of the nucleotide sequence 5′-asdGsucdAudAaaaudTcAfggaauucscsu-3′ of SEQ ID NO:1922,
wherein a, g, c and u are 2′-O-methyl (2′-OMe) A, G, C, and U, respectively; Af and Gf are 2′-fluoro A and G, respectively; dG is a 2′-deoxyguanosine-3′-phosphate nucleotide; dA is a 2′-deoxyadenosine-3′-phosphate nucleotide; dT is a 2′-deoxythymidine-3′-phosphate nucleotide; and s is a phosphorothioate linkage, and
wherein the 3′-end of the sense strand is conjugated to a ligand as shown in the following schematic, , wherein X is O.