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US 2026/0234624 A1Published application
Compositions and methods for modulating ttr expression
AssigneeIonis Pharmaceuticals, Inc.
InventorsThazha P. PRAKASH, Punit P. SETH, Eric E. SWAYZE
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Abstract
Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.
Claims16
-42. (canceled)
A compound represented by the chemical structure:
- (SEQ ID NO:
- 12), or a pharmaceutically acceptable salt thereof,
- wherein either R1 is —OCH2CH2OCH3 (MOE) and R2 is H;
- or R1 and R2 together form a bridge, wherein R1 is —O— and R2 is —CH2—, —CH(CH3)—, or —CH2CH2—, and R1 and R2 are directly connected such that the resulting bridge is selected from:
- —O—CH2—, —O—CH(CH3)—, and —O—CH2CH2—;
- and for each pair of R3 and R4 on the same ring, independently for each ring:
- either R3 is selected from H and —OCH2CH2OCH3 and R4 is H;
- or R3 and R4 together form a bridge, wherein R3 is —O—, and R4 is —CH2—, —CH(CH3)—, or —CH2CH2— and R3 and R4 are directly connected such that the resulting bridge is selected from:
- —O—CH2—, —O—CH(CH3)—, and —O—CH2CH2—;
- and R5 is selected from H and —CH3;
- and Z is selected from S− and O−.
8 more elements
Dependencies are inferred from the claim text.