US 12,331,293 B2
Bispecific antisense oligonucleotides for dystrophin exon skipping
Judith Christina Theodora van Deutekom, Dordrecht (NL); and Nicole Anne Datson, Oegstgeest (NL)
Assigned to BIOMARIN TECHNOLOGIES B.V., Amsterdam (NL)
Appl. No. 17/309,140
Filed by BioMarin Technologies B.V., Amsterdam (NL)
PCT Filed Oct. 30, 2019, PCT No. PCT/EP2019/079714
§ 371(c)(1), (2) Date Apr. 29, 2021,
PCT Pub. No. WO2020/089325, PCT Pub. Date May 7, 2020.
Claims priority of application No. 18204170 (EP), filed on Nov. 2, 2018.
Prior Publication US 2022/0025368 A1, Jan. 27, 2022
Int. Cl. C12N 15/113 (2010.01); A61P 21/00 (2006.01)
CPC C12N 15/113 (2013.01) [A61P 21/00 (2018.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/51 (2013.01); C12N 2320/33 (2013.01)] 15 Claims
OG exemplary drawing
 
1. A compound comprising a first and a second antisense oligonucleotide (AON) linked to each other by a linking moiety, wherein said first antisense oligonucleotide (AON) consists of the base sequence of SEQ ID NO: 14, and wherein said second antisense oligonucleotide (AON) consists of the base sequence of SEQ ID NO: 198, wherein sequences complementary to SEQ ID NO: 14 and 198 are located within exon 51 of dystrophin pre-mRNA.