IP Library Patent Application 15896379
Patent Application
App. No. 15/896,379

COMPOSITIONS AND METHODS FOR MODULATING HBV EXPRESSION

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Patent No.
US None
App. No.
15/896,379
Abstract

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

Claims (35)

1 - 25 . (canceled)

26 . A compound comprising a modified oligonucleotide and a conjugate group, wherein the modified oligonucleotide consists of 16 linked nucleosides having a nucleobase sequence consisting of the nucleobase sequence of SEQ ID NO: 10, and wherein the conjugate group comprises:

27 . The compound of claim 26 , wherein the modified oligonucleotide comprises at least one modified sugar.

28 . The compound of claim 27 , wherein at least one modified sugar is a bicyclic sugar.

29 . The compound of claim 27 , wherein at least one modified sugar comprises a 2′-O-methoxyethyl, a constrained ethyl, or a 4′-(CH 2 ) n —O-2′ bridge, wherein n is 1 or 2.

30 . The compound of claim 27 , wherein at least one modified sugar is 2′-O-methoxyethyl.

31 . The compound of claim 26 , wherein at least one nucleoside comprises a modified nucleobase.

32 . The compound of claim 31 , wherein the modified nucleobase is a 5-methylcytosine.

33 . The compound of claim 26 , wherein the conjugate group is linked to the modified oligonucleotide at the 5′ end of the modified oligonucleotide.

34 . The compound of claim 26 , wherein the conjugate group is linked to the modified oligonucleotide at the 3′ end of the modified oligonucleotide.

35 . The compound of claim 26 , wherein each internucleoside linkage of the modified oligonucleotide is selected from a phosphodiester internucleoside linkage and a phosphorothioate internucleoside linkage.

36 . The compound of claim 35 , wherein the modified oligonucleotide comprises at least 5 phosphodiester internucleoside linkages.

37 . The compound of claim 35 , wherein the modified oligonucleotide comprises at least two phosphorothioate internucleoside linkages.

38 . The compound of claim 26 , wherein the modified oligonucleotide is single-stranded.

39 . The compound of claim 26 , wherein the modified oligonucleotide is double stranded.

40 . The compound of claim 26 , wherein the modified oligonucleotide comprises:

a gap segment consisting of linked deoxynucleosides;

a 5′ wing segment consisting of linked nucleosides;

a 3′ wing segment consisting of linked nucleosides;

wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment and wherein each nucleoside of each wing segment comprises a modified sugar.

41 . The compound of claim 40 , wherein each internucleoside linkage of the modified oligonucleotide is selected from a phosphodiester internucleoside linkage and a phosphorothioate internucleoside linkage.

42 . The compound of claim 41 , wherein each internucleoside linkage in the gap segment of the modified oligonucleotide is a phosphorothioate linkage.

43 . The compound of claim 42 , wherein the modified oligonucleotide further comprises at least one phosphorothioate internucleoside linkage in each wing segment.

44 . The compound of claim 43 , wherein the modified sugar is a bicyclic sugar.

45 . The compound of claim 43 , wherein the modified sugar comprises a 2′-O-methoxyethyl, a constrained ethyl, or a 4′-(CH 2 ) n —O-2′ bridge, wherein n is 1 or 2.

46 . The compound of claim 43 , wherein the modified sugar is 2′-O-methoxyethyl.

47 . The compound of claim 43 , wherein the modified sugar of each wing segment comprises at least one 2′-O-methoxyethyl and at least one constrained ethyl.

48 . The compound of claim 47 , wherein at least one nucleoside comprises a modified nucleobase.

49 . The compound of claim 48 , wherein the modified nucleobase is a 5-methylcytosine.

50 . The compound of claim 49 , wherein the conjugate group is linked to the modified oligonucleotide at the 5′ end of the modified oligonucleotide.

51 . The compound of claim 26 , wherein the modified oligonucleotide comprises:

a gap segment consisting of 10 linked deoxynucleosides;

a 5′ wing segment consisting of 2 linked nucleosides; and

a 3′ wing segment consisting of 4 linked nucleosides;

wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment; wherein the 2 linked nucleosides of the 5′ wing segment comprise a 2′-O-methoxyethyl sugar and a constrained ethyl sugar in the 5′ to 3′ direction; wherein the 4 linked nucleosides of the 3′ wing segment comprise a constrained ethyl sugar, a 2′-O-methoxyethyl sugar, a constrained ethyl sugar, and a 2′-O-methoxyethyl sugar in the 5′ to 3′ direction; and wherein each cytosine residue is a 5-methylcytosine.

Assignments (2)
CORRECTIVE ASSIGNMENT TO CORRECT THE ASSIGNMENT PREVIOUSLY RECORDED IN ERROR AND REMOVE ALL PROPERTY PREVIOUSLY RECORDED ON REEL 062568 FRAME 0001. ASSIGNOR(S) HEREBY CONFIRMS THE ASSIGNMENT. Recorded Dec 22, 2023
From: IONIS PHARMACEUTICALS, INC.
To: IONIS PHARMACEUTICALS, INC
Reel/Frame 066126/0812 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 31, 2023
From: IONIS PHARMACEUTICALS, INC.
To: GLAXO GROUP LIMITED
Reel/Frame 062568/0001 →