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US 2026/0263605 A1Published application
Compound, conjugate, composition, and uses thereof
AssigneeRigerna Therapeutics (Beijing) Co., Ltd.
InventorsYuanyu HUANG, Xiaofeng HAN
Life of this patent
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Abstract
The present disclosure relates to a compound, a conjugate, a composition, and uses thereof. Specifically, the present disclosure relates to a compound having a structure represented by specific formula (Ia), a stereoisomer, pharmaceutically acceptable salt, or prodrug thereof. The compound can be used for preventing and/or treating the expression or activity of a specific target gene.
Claims21
A compound represented by formula (Ia), a stereoisomer, a pharmaceutically acceptable salt, or a prodrug thereof,
- in formula (Ia), R1 represents a hydroxyl protecting group,
- R2 is selected from the group consisting of:
- wherein
- represents a solid phase support, and R2′ is a covalent linking group connected to the solid phase support,
- n is selected from the group consisting of 0, 1, 2 and 3,
- each Z is independently selected from the group consisting of hydroxyl and mercapto,
- each p is independently selected from the group consisting of 1, 2 and 3,
- each q is independently selected from the group consisting of 1, 2 and 3,
- each A is independently an unsubstituted or substituted 4- to 10-membered aliphatic ring,
- each X is independently selected from the group consisting of NH, O and S,
- each L1 is independently selected from the group consisting of
- wherein j is selected from the group consisting of 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10,
- each R3 is independently selected from the group consisting of H, C1-C6 alkyl, C1-C6 haloalkyl, and C1-C6 alkoxy,
- each L2 is independently selected from the group consisting of C1-C30 alkylidene and
- wherein each RL2a is independently C1-C10 alkylidene, each RL2b is independently selected from the group consisting of O, S, NH, and —NH—C(O)—, and k is selected from the group consisting of 1, 2, 3, 4, 5, 6, 7, 8, 9, and 10,
- each Y is independently selected from the group consisting of NH, O and S, and
- each R4 is independently selected from the group consisting of:
14 more elements
(canceled)
Dependencies are inferred from the claim text.