US 12,359,201 B2
17ß-hydroxysteroid dehydrogenase type 13 (HSD17B13) iRNA compositions and methods of use thereof
Gregory Hinkle, Plymouth, MA (US); Frederic Tremblay, Boston, MA (US); Noura S. Abul-Husn, New York, NY (US); Omri Gottesman, Rancho Santa Fe, CA (US); Alexander Li, White Plains, NY (US); Xiping Cheng, Northvale, NJ (US); Yurong Xin, Ashland, MA (US); Jesper Gromada, Concord, MA (US); Frederick E. Dewey, Winchester, MA (US); Aris Baras, Bedford Corners, NY (US); Alan Shuldiner, Baltimore, MD (US); and Stuart Milstein, Arlington, MA (US)
Assigned to Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
Appl. No. 16/978,947
Filed by Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
PCT Filed Mar. 20, 2019, PCT No. PCT/US2019/023079
§ 371(c)(1), (2) Date Sep. 8, 2020,
PCT Pub. No. WO2019/183164, PCT Pub. Date Sep. 26, 2019.
Claims priority of provisional application 62/775,590, filed on Dec. 5, 2018.
Claims priority of provisional application 62/770,298, filed on Nov. 21, 2018.
Claims priority of provisional application 62/645,941, filed on Mar. 21, 2018.
Prior Publication US 2021/0380985 A1, Dec. 9, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/113 (2010.01)
CPC C12N 15/113 (2013.01) [C12N 15/1137 (2013.01); C12N 2310/11 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/3515 (2013.01); C12N 2310/3521 (2013.01); C12N 2310/3533 (2013.01); C12N 2320/32 (2013.01); C12N 2320/50 (2013.01)] 39 Claims
 
1. A method of inhibiting 17β-hydroxysteroid dehydrogenase type 13 (HSD17B13) expression in a cell, comprising contacting the cell with a double stranded ribonucleic acid (dsRNA) agent, wherein the dsRNA agent comprises a sense strand and an antisense strand, wherein the nucleotide sequence of the sense strand comprises the nucleotide sequence of SEQ ID NO: 4498 and the nucleotide sequence of the antisense strand comprises the nucleotide sequence of SEQ ID NO: 4499, thereby inhibiting expression of HSD17B13 in the cell.