US 12,473,550 B2
Coagulation factor V (F5) iRNA compositions and methods of use thereof
Mark Keating, Weston, MA (US); James D. McIninch, Burlington, MA (US); and Mark K. Schlegel, Boston, MA (US)
Assigned to Alnylam Pharmaceuticals, Inc., Cambridge, MA (US)
Filed by Alnylam Pharmaceuticals, Inc., Cambridge, MA (US)
Filed on May 8, 2023, as Appl. No. 18/144,276.
Application 18/144,276 is a continuation of application No. PCT/US2021/059047, filed on Nov. 12, 2021.
Claims priority of provisional application 63/271,872, filed on Oct. 26, 2021.
Claims priority of provisional application 63/146,115, filed on Feb. 5, 2021.
Claims priority of provisional application 63/113,282, filed on Nov. 13, 2020.
Prior Publication US 2024/0209358 A1, Jun. 27, 2024
Int. Cl. C12N 15/113 (2010.01); A61K 9/00 (2006.01); A61K 45/06 (2006.01); A61K 47/54 (2017.01); A61P 7/02 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 9/0019 (2013.01); A61K 45/06 (2013.01); A61K 47/549 (2017.08); A61P 7/02 (2018.01); C12N 2310/11 (2013.01); C12N 2310/3125 (2013.01); C12N 2310/313 (2013.01)] 55 Claims
 
1. A method of inhibiting expression of a coagulation Factor V (F5) gene in a cell, the method comprising contacting the cell with a double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of coagulation Factor V (F5) in a cell, or a pharmaceutically acceptable salt thereof,
wherein the dsRNA agent, or a pharmaceutically acceptable salt thereof, comprises a sense strand differing by no more than 4 unmodified or modified bases from the nucleotide sequence 5′-ascsaguuuuCfCfAfcuauuucucu-3′ of SEQ ID NO:2739 and an antisense strand differing by no more than 4 unmodified or modified bases from the nucleotide sequence 5′-asdGsagdAadAuagudGgAfaaacugususa-3′ of SEQ ID NO:2940,
wherein a, g, c and u are 2′-O-methyl (2′-OMe) A, G, C, and U, respectively; Af and Cf, are 2′-fluoro A and C, respectively; s is a phosphorothioate linkage; dG is 2-deoxyguanosine-3′-phosphate; and dA is 2-deoxyadenosine-3-phosphate, thereby inhibiting expression of the F5 gene in the cell.