	US 12,071,626 B2
	Splice-switching oligonucleotides and methods of use
Jennifer Freedman, Durham, NC (US); Brendon Patierno, Durham, NC (US); Bonnie LaCroix, Durham, NC (US); Timothy Robinson, Durham, NC (US); Bruce Sullenger, Durham, NC (US); Daniel George, Durham, NC (US); and Steven Patierno, Durham, NC (US)
Assigned to Duke University, Durham, NC (US)
Filed by Duke University, Durham, NC (US)
Filed on Sep. 1, 2021, as Appl. No. 17/464,474.
Application 17/464,474 is a continuation of application No. 15/773,841, granted, now 11,136,584, previously published as PCT/US2016/060549, filed on Nov. 4, 2016.
Claims priority of provisional application 62/274,427, filed on Jan. 4, 2016.
Claims priority of provisional application 62/250,713, filed on Nov. 4, 2015.
Prior Publication US 2021/0395754 A1, Dec. 23, 2021
Int. Cl. C12N 15/11 (2006.01); A61K 31/7105 (2006.01); A61K 31/7125 (2006.01); A61K 45/06 (2006.01); A61P 35/00 (2006.01); C07H 21/04 (2006.01); C12N 15/113 (2010.01); A61K 48/00 (2006.01)

CPC C12N 15/1138 (2013.01) [A61K 31/7105 (2013.01); A61K 31/7125 (2013.01); A61K 45/06 (2013.01); A61P 35/00 (2018.01); C07H 21/04 (2013.01); C12N 15/11 (2013.01); C12N 15/1136 (2013.01); A61K 48/0066 (2013.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2320/31 (2013.01); C12N 2320/33 (2013.01)]

29 Claims

1. A modified splice-switching oligonucleotide (SSO), said SSO comprising:

(i) a nucleotide sequence set forth in SEQ ID NO: 6,

(ii) a length of at least 18 nucleotides and no more than 100 nucleotides,

(iii) full complementarity to a sequence within exon 18 (SEQ ID NO: 8) of an EGFR pre-mRNA, and

(iv) a modification selected from the group consisting of a 2′-O-methyl phosphorothioate, a morpholino, a phosphorodiamidate-linked morpholino (PMO), a locked nucleic acid (LNA), a peptide nucleic acid, a 2′-O-(2-methoxy ethyl) nucleotide, a G-clamp or 9-(aminoethoxy)phenoxazine nucleotide, and cytosine analogues that form 4 hydrogen bonds with guanosine.