US 11,912,997 B2
RNAi agents for inhibiting expression of Superoxide Dismutase 1 (SOD1), compositions thereof, and methods of use
Christine Esau, San Diego, CA (US); Ji Young Suk, San Diego, CA (US); Tao Pei, Middleton, WI (US); Anthony Nicholas, Oregon, WI (US); Xiaokai Li, Middleton, WI (US); and Jeffrey Carlson, Madison, WI (US)
Assigned to Arrowhead Pharmaceuticals, Inc., Pasadena, CA (US)
Filed by Arrowhead Pharmaceuticals, Inc., Pasadena, CA (US)
Filed on Jun. 14, 2023, as Appl. No. 18/334,932.
Claims priority of provisional application 63/495,517, filed on Apr. 11, 2023.
Claims priority of provisional application 63/352,454, filed on Jun. 15, 2022.
Prior Publication US 2024/0026363 A1, Jan. 25, 2024
Int. Cl. C12N 15/113 (2010.01); A61P 35/00 (2006.01); A61P 25/28 (2006.01)
CPC C12N 15/1137 (2013.01) [A61P 25/28 (2018.01); C12N 2310/11 (2013.01); C12N 2310/32 (2013.01); C12N 2310/3515 (2013.01)] 30 Claims
 
1. An RNAi agent for inhibiting expression of a Superoxide Dismutase 1 (SOD1) gene, comprising:
an antisense strand comprising the nucleotide sequence cPrpusGfsaGfaucacagAfaUfcUfucasasc (SEQ ID NO: 646); and
a sense strand comprising the nucleotide sequence guugaagaUfuCfuGfugaucuca (SEQ ID NO: 771)
wherein a, c, g, and u represent 2′-O-methyl adenosine, 2′-O-methyl cytidine, 2′-O-methyl guanosine, and 2′-O-methyl uridine, respectively; Af, Cf, Gf, and Uf represent 2′-fluoro adenosine, 2′-fluoro cytidine, 2′-fluoro guanosine, and 2′-fluoro uridine, respectively; cPrpu represents a 5′-cyclopropyl phosphonate-2′-O-methyl uridine, and s represents a phosphorothioate linkage.