Antisense oligonucleotide composite
JP2024015491A5Pending Publication Date: 2025-10-03LIID PHARM INC
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Patent Information
- Application Number
- JP2023200811
- Authority / Receiving Office
- JP · JP
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2021-12-07
- Filing Date
- 2023-11-28
- Publication Date
- 2025-10-03
AI Technical Summary
Technical Problem
Existing antisense oligonucleotides face challenges with off-target toxicity due to hybridization-dependent and hybridization-independent mechanisms, hindering their clinical use and expansion of indications.
Method used
A novel antisense oligonucleotide complex, BROTHERS, is developed with a toehold region and a complementary strand that forms a well-controlled higher-order structure, reducing toxicity by minimizing hybridization-dependent effects and enhancing nuclease resistance.
Benefits of technology
BROTHERS effectively suppresses hepatotoxicity and nephrotoxicity while maintaining antisense activity, demonstrating reduced toxicity and efficient gene expression regulation in target cells.
✦ Generated by Eureka AI based on patent content.
Abstract
To provide a novel antisense oligonucleotide composite with reduced toxicity while having activity of antisense oligonucleotide, a pharmaceutical composition containing the antisense oligonucleotide composite, and a composition for regulating expression of a target gene which contains the antisense composite.SOLUTION: An antisense oligonucleotide composite having an antisense effect and reduced toxicity contains antisense oligonucleotide, and a complementary strand including a sequence complementary with the antisense oligonucleotide, where the antisense oligonucleotide has a toehold of a single-stranded structure, and the complementary strand has nuclease tolerance over the entire length.SELECTED DRAWING: None
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