	US 12,485,136 B2
	Methods for the treatment of trinucleotide repeat expansion disorders associated with MLH3 activity
Nessan Anthony Bermingham, Cambridge, MA (US); Brian R. Bettencourt, Cambridge, MA (US); and Peter Edward Bialek, Cambridge, MA (US)
Assigned to Takeda Pharmaceuticals U.S.A., Inc., Cambridge, MA (US)
Appl. No. 17/299,277
Filed by TAKEDA PHARMACEUTICALS U.S.A., INC., Cambridge, MA (US)
PCT Filed Dec. 2, 2019, PCT No. PCT/US2019/064059 § 371(c)(1), (2) Date Jun. 2, 2021, PCT Pub. No. WO2020/117706, PCT Pub. Date Jun. 11, 2020.
Claims priority of provisional application 62/877,104, filed on Jul. 22, 2019.
Claims priority of provisional application 62/774,777, filed on Dec. 3, 2018.
Prior Publication US 2023/0042436 A1, Feb. 9, 2023
Int. Cl. A61K 31/7125 (2006.01); A61P 25/28 (2006.01); C12N 15/113 (2010.01)

CPC A61K 31/7125 (2013.01) [A61P 25/28 (2018.01); C12N 15/113 (2013.01)]

9 Claims

1. A single-stranded oligonucleotide of 20 linked nucleosides wherein the nucleobase sequence consists of SEQ ID NO: 515, 1302, or 2491, wherein all thymidines at positions 1-5 and 16-20 of the nucleobase sequence are substituted with 5-methyl-2′-O-methoxyethyl-uridine and all cytidines at positions 6-15 are substituted with 5-methyl-2′-O-methoxyethyl-cytidine, and wherein the backbone is fully phosphorothioate-modified.