US 11,866,710 B2
Transmembrane protease, serine 6 (TMPRSS6) iRNA compositions and methods of use thereof
Aimee M. Deaton, Somerville, MA (US); John Michael Gansner, Newton, MA (US); James D. McIninch, Burlington, MA (US); Mark K. Schlegel, Boston, MA (US); and Benjamin P. Garfinkel, Brookline, MA (US)
Assigned to Alnylam Pharmaceuticals, Inc., Cambridge, MA (US)
Filed by Alnylam Pharmaceuticals, Inc., Cambridge, MA (US)
Filed on Jan. 6, 2023, as Appl. No. 18/150,827.
Application 18/150,827 is a continuation of application No. PCT/US2022/026097, filed on Apr. 25, 2022.
Claims priority of provisional application 63/278,227, filed on Nov. 11, 2021.
Claims priority of provisional application 63/179,607, filed on Apr. 26, 2021.
Prior Publication US 2023/0220396 A1, Jul. 13, 2023
Int. Cl. C07H 21/04 (2006.01); C12N 15/113 (2010.01); A61K 47/54 (2017.01)
CPC C12N 15/1137 (2013.01) [A61K 47/549 (2017.08); C12Y 304/21109 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/351 (2013.01)] 46 Claims
 
1. A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of Transmembrane protease, serine 6 (TMPRSS6) in a cell, or a pharmaceutically acceptable salt thereof, comprising a sense strand and an antisense strand forming a double stranded region,
wherein the nucleotide sequence of the sense strand differs by no more than 4 bases from the nucleotide sequence 5′-asgscugcccUfUfUfggaauaaagu-3′ (SEQ ID NO:395) and the nucleotide sequence of the antisense strand differs by no more than 4 bases from the nucleotide sequence 5′-asdCsuudTadTuccadAaGfggcagcusgsa-3′ (SEQ ID NO:521),
wherein a, g, c and u are 2′-O-methyl (2′-OMe) A, G, C, and U, respectively; Gf and Uf are 2′-deoxy-2′-fluoro (2′-F) G and U, respectively; dC, dA, and dT are 2′-deoxy C, A, and T, respectively; and s is a phosphorothioate linkage, and
wherein the dsRNA agent is conjugated to a ligand.