Compositions and methods for treating muscle atrophy and myotonic dystrophies
JP2025000818A5Pending Publication Date: 2025-03-27AVIDITY BIOSCI INC
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Patent Information
- Application Number
- JP2024169131
- Authority / Receiving Office
- JP · JP
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2018-08-31
- Filing Date
- 2024-09-27
- Publication Date
- 2025-03-27
Abstract
To provide compositions and methods for treating muscle atrophy and myotonic dystrophies.SOLUTION: This application discloses a polynucleic acid molecule conjugate comprising an antibody or binding fragment thereof conjugated to a polynucleic acid molecule that hybridizes to a target sequence of an atrogene, wherein the polynucleic acid molecule comprises at least one 2' modified nucleotide, at least one modified internucleotide linkage, or at least one inverted abasic moiety, and the polynucleic acid molecule conjugate mediates RNA interference against the atrogene, thereby treating muscle atrophy or myotonic dystrophy in a subject. In a certain embodiment, atrogene comprises atrogin-1 gene (FBXO32), MuRF1 gene (TRIA63), FOXO1, FOXO3, or MSTN.SELECTED DRAWING: Figure 19A
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