	US 11,911,403 B2
	Antisense-induced exon exclusion in type VII collagen
Dan V. Mourich, Cambridge, MA (US)
Assigned to Sarepta Therapeutics, Inc., Cambridge, MA (US)
Filed by Sarepta Therapeutics, Inc., Cambridge, MA (US)
Filed on Oct. 26, 2020, as Appl. No. 17/080,363.
Application 17/080,363 is a continuation of application No. 15/578,612, granted, now 10,849,917, previously published as PCT/US2016/035326, filed on Jun. 1, 2016.
Claims priority of provisional application 62/169,454, filed on Jun. 1, 2015.
Prior Publication US 2021/0161922 A1, Jun. 3, 2021
Int. Cl. C07H 21/02 (2006.01); A61K 31/70 (2006.01); C12N 15/113 (2010.01); C07H 21/04 (2006.01); A61P 17/00 (2006.01)

CPC A61K 31/70 (2013.01) [A61P 17/00 (2018.01); C07H 21/02 (2013.01); C07H 21/04 (2013.01); C12N 15/113 (2013.01); C12N 2310/11 (2013.01); C12N 2310/3145 (2013.01); C12N 2310/3181 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/3233 (2013.01); C12N 2320/33 (2013.01)]

8 Claims

1. An antisense oligomer compound of 12 to 40 subunits, comprising:

at least one subunit that is a nucleotide analog having (i) a modified internucleoside linkage, (ii) a modified sugar moiety, or (iii) a combination of the foregoing; and

a targeting sequence complementary to 12 or more contiguous nucleotides in a target region spanning an exon/intron junction of human type VII collagen pre-mRNA, wherein the contiguous nucleotides include the exon/intron junction, and wherein said exon/intron junction comprises the splice junction of exon 80/intron 80, wherein the targeting sequence is selected from SEQ ID NOS: 27, 28, 29, and 30.