Patent ID: 9550988
Date: 2017-01-24
CPC Classifications: A61K,A61P,C12N

Claim:
1. A gapmer oligonucleotide consisting of 10 to 24 linked nucleosides wherein the gapmer oligonucleotide consists of three regions: a gap region consisting of 6 to 10 linked deoxynucleosides; a 5′ wing region positioned at the 5′ end of the gap region and consisting of 1 to 7 linked nucleosides, wherein each nucleoside of the 5′ wing region comprises a modified sugar, and wherein at least one nucleoside of the 5′ wing region is a 2′-O(CH a 3′ wing region positioned at the 3′ end of the gap region and consisting of 1 to 7 linked nucleosides, wherein each nucleoside of the 3′ wing region comprises a modified sugar, and wherein at least one nucleoside of the 3′ wing region is a 4′ to 2′ bicyclic nucleoside; and wherein a plurality of the internucleoside linkages of the gapmer oligonucleotide are phosphorothioate internucleoside linkages; or a pharmaceutically acceptable salt thereof.