US 12,391,942 B2
Oligonucleotide compositions and methods of use thereof
Jason Jingxin Zhang, Walpole, MA (US); Chandra Vargeese, Schwenksville, PA (US); Naoki Iwamoto, Boston, MA (US); Chikdu Shakti Shivalila, Woburn, MA (US); Nayantara Kothari, Newton, MA (US); Ann Fiegen Durbin, Arlington, MA (US); Selvi Ramasamy, Wayland, MA (US); Pachamuthu Kandasamy, Belmont, MA (US); Jayakanthan Kumarasamy, Belmont, MA (US); Gopal Reddy Bommineni, Belmont, MA (US); Subramanian Marappan, Acton, MA (US); Sethumadhavan Divakaramenon, Lexington, MA (US); David Charles Donnell Butler, Medford, MA (US); Genliang Lu, Winchester, MA (US); Hailin Yang, Brighton, MA (US); Mamoru Shimizu, Arlington, MA (US); and Prashant Monian, Arlington, MA (US)
Assigned to WAVE LIFE SCIENCES LTD., Singapore (SG)
Appl. No. 17/054,452
Filed by WAVE LIFE SCIENCES LTD., Singapore (SG)
PCT Filed May 10, 2019, PCT No. PCT/US2019/031672
§ 371(c)(1), (2) Date Nov. 10, 2020,
PCT Pub. No. WO2019/217784, PCT Pub. Date Nov. 14, 2019.
Claims priority of provisional application 62/776,432, filed on Dec. 6, 2018.
Claims priority of provisional application 62/723,375, filed on Aug. 27, 2018.
Claims priority of provisional application 62/715,684, filed on Aug. 7, 2018.
Claims priority of provisional application 62/670,709, filed on May 11, 2018.
Claims priority of application No. PCT/US2019/027109 (WO), filed on Apr. 11, 2019.
Prior Publication US 2021/0254062 A1, Aug. 19, 2021
Int. Cl. C12N 15/113 (2010.01)
CPC C12N 15/113 (2013.01) [C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2320/33 (2013.01); C12N 2330/30 (2013.01)] 32 Claims
 
1. An oligonucleotide, wherein the oligonucleotide is:
fC * SfU * SfCn001RfC * SfG * SfGn001RfU * SfU * SmCfU * SmG * SfA * SmAfG * SfG * SfU * SfGn001RfU * SfU * SfC (SEQ ID NO: 11)or a pharmaceutically acceptable salt thereof, wherein:
f represents a 2′—F modified nucleoside;
* S represents a Sp phosphorothioate;
m represents a 2′-OMe modified nucleoside; and
n001R is

OG Complex Work Unit Chemistry
 wherein the phosphorus is of the Rp configuration.