US 12,234,447 B2
Modified compounds and uses thereof
Punit P. Seth, Carlsbad, CA (US); Michael Oestergaard, Carlsbad, CA (US); Michael T. Migawa, Carlsbad, CA (US); Xue-Hai Liang, Del Mar, CA (US); Wen Shen, Carlsbad, CA (US); Stanley T. Crooke, Carlsbad, CA (US); and Eric E. Swayze, Encinitas, CA (US)
Assigned to Ionis Pharmaceuticals, Inc., Carlsbad, CA (US)
Filed by Ionis Pharmaceuticals, Inc., Carlsbad, CA (US)
Filed on Mar. 17, 2022, as Appl. No. 17/697,577.
Application 17/697,577 is a continuation of application No. 16/968,701, granted, now 11,332,733, previously published as PCT/US2019/017725, filed on Feb. 12, 2019.
Prior Publication US 2023/0068063 A1, Mar. 2, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. C07H 21/04 (2006.01); C12N 15/10 (2006.01); C12N 15/113 (2010.01)
CPC C12N 15/10 (2013.01) [C12N 15/113 (2013.01); C12N 2310/315 (2013.01); C12N 2310/3231 (2013.01)] 21 Claims
 
1. An oligomeric compound comprising a modified oligonucleotide consisting of 16-20 linked nucleosides, wherein the modified oligonucleotide has a 5′-region, a central region, and a 3′-region, wherein:
the 5′-region consists of 3-5 linked nucleosides, each comprising modified sugar moiety;
the 3′ region consists of 3-5 linked nucleosides, each comprising a modified sugar moiety;
and the central region consists of 8-10 linked nucleosides, wherein the central region has the following formula:
(Nd)(Nx)(Nd)n
wherein Nx is a nucleoside comprising a 2′-OMe-β-D-ribofuranosyl sugar moiety and each Nd is a nucleoside comprising a 2′-β-D-deoxyribosyl sugar moiety;
and n is 6-8.