Therapeutic agents for metabolism-associated diseases
AssigneeUnited Kingdom Research and Innovation
InventorsSantiago VERNIA, Helen PATERSON, Sijia YU, Michael MANOLAKAKIS, Andrew JOBBINS
Life of this patent
Estimating the term…
Abstract
Provided herein is a target for the treatment of metabolism-associated diseases, such as metabolism-associated fatty liver disease (MAFLD). Also provided are agents for the treatment of said diseases and methods of using said agents.
Claims34
An antisense oligomer capable of inducing skipping of exon 12 of scavenger receptor class B type I (SCARB1).
The antisense oligomer of any preceding claim, wherein the antisense oligomer is complementary to 5, 10, 15, 16, 17, 18, 19, 20, or all bases to which an antisense oligomer according to any one of SEQ ID NOs:
- 4, 5, 7, 8, 9, 10, 11, 12, 18, or 19 is complementary.
The antisense oligomer of any preceding claim, wherein the antisense oligomer is complementary to 5, 10, 15, 16, 17, 18, 19, or all 20 bases to which SSO8.18 (SEQ ID NO:
- 51) is complementary.
The antisense oligomer of any preceding claim, wherein the antisense oligomer comprises at least one 2′-O-methyl nucleotide and/or at least one 2′-O-methoxyethyl nucleotide.
The antisense oligomer of any preceding claim, wherein the antisense oligomer is a 2′-O-methyl nucleic acid oligomer and/or a 2′-O-methoxyethyl nucleic acid oligomer.
The antisense oligomer of any preceding claim, wherein the antisense oligomer comprises at least one phosphorothioate internucleotide linkage.
The antisense oligomer of any preceding claim, wherein all internucleotide linkages in the antisense oligomer are phosphorothioate internucleotide linkages.
The antisense oligomer of any preceding claim, wherein the antisense oligomer is an oligonucleotide and has a nucleobase sequence according to SEQ ID NO:
- 5, 8, 10, 11, 18, or 19, and wherein —O—CH3 or —O—CH2—CH2—O—CH3 is attached to the 2′ position of the sugar moiety of each nucleotide.
An antisense oligonucleotide with a sequence according to any one of SEQ ID NOs:
- 4, 5, 7, 8, 9, 10, 11, 12, 18, or 19, wherein the internucleotide linkages are phosphorothioate internucleotide linkages, and wherein —O—CH3 or —O—CH2—CH2—O—CH3 is attached to the 2′ position of the sugar moiety of each nucleotide.
The antisense oligomer of any preceding claim, wherein the antisense oligomer is or comprises SSO8.5 (SEQ ID NO:
- 38), SSO8.8 (SEQ ID NO:
- 41), SSO8.10 (SEQ ID NO:
- 43), SSO8.11 (SEQ ID NO:
- 44), SSO8.18 (SEQ ID NO:
- 51), SSO8.19 (SEQ ID NO:
- 52), or SSO8.21 (SEQ ID NO:
- 54).
The antisense oligomer of any preceding claim, wherein the antisense oligomer is or comprises SSO8.18 (SEQ ID NO:
- 51).
Dependencies are inferred from the claim text.