US 11,866,689 B2
Splice-switching oligonucleotides and methods of use
Jennifer Freedman, Durham, NC (US); Steven Patierno, Durham, NC (US); Daniel George, Durham, NC (US); Bruce Sullenger, Durham, NC (US); Bonnie LaCroix, Durham, NC (US); and Brendon Patierno, Durham, NC (US)
Assigned to Duke University, Durham, NC (US)
Appl. No. 17/041,843
Filed by Duke University, Durham, NC (US)
PCT Filed Mar. 22, 2019, PCT No. PCT/US2019/023670
§ 371(c)(1), (2) Date Sep. 25, 2020,
PCT Pub. No. WO2019/190927, PCT Pub. Date Oct. 3, 2019.
Claims priority of provisional application 62/647,897, filed on Mar. 26, 2018.
Prior Publication US 2021/0024932 A1, Jan. 28, 2021
Int. Cl. C07H 21/04 (2006.01); C12N 15/113 (2010.01); A61K 31/4166 (2006.01); A61K 31/712 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 31/4166 (2013.01); A61K 31/712 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/33 (2013.01)] 22 Claims
 
1. An anti-sense compound comprising a modified splice-switching oligonucleotide (SSO) consisting of 22 to 30 linked nucleosides and having a nucleobase sequence at least 80% complementary to the corresponding nucleotide sequence within SEQ ID NO:5, wherein the SSO comprises a sequence selected from the group consisting of SEQ ID NO:7, SEQ ID NO:9, SEQ ID NO: 11 and SEQ ID NO: 13, and a sequence with at least 80% identity to SEQ ID NO:7, SEQ ID NO:9, SEQ ID NO: 11 or SEQ ID NO: 13, wherein each nucleoside of the SSO comprises a sugar modification.