Double-stranded ribonucleic acid targeting PNPLA3
Modified dsRNA targeting PNPLA3 with 2'-F and 2'-deoxy modifications addresses the degradation issues of unmodified RNAi agents, offering a stable and safe treatment for NASH by inhibiting PNPLA3 expression and reducing liver damage.
TW202627094APending Publication Date: 2026-07-01CHIA TAI TIANQING PHARMA GRP CO LTD
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Patent Information
- Application Number
- TW114150287
- Authority / Receiving Office
- TW · TW
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2025-12-16
- Filing Date
- 2025-12-19
- Publication Date
- 2026-07-01
AI Technical Summary
Technical Problem
Unmodified RNAi reagents exhibit poor metabolic stability and are readily degraded in vivo, necessitating the development of chemically modified RNAi agents with enhanced efficacy, stability, reduced toxicity, and improved safety for treating nonalcoholic steatohepatitis (NASH) by targeting the PNPLA3 gene.
Method used
Development of double-stranded ribonucleic acid (dsRNA) with specific nucleotide modifications, including 2'-F and 2'-deoxy modifications, designed to inhibit PNPLA3 expression, thereby regulating lipolysis and reducing liver damage associated with NASH.
Benefits of technology
The modified dsRNA effectively inhibits PNPLA3 expression, enhancing metabolic stability and safety, providing a potential therapeutic approach for NASH with reduced toxicity.
✦ Generated by Eureka AI based on patent content.
Abstract
The present application belongs to the field of biomedicine, and relates to a double-stranded ribonucleic acid (dsRNA) targeting PNPLA3, a pharmaceutically acceptable salt thereof, a ligand conjugate thereof, or a pharmaceutical composition.
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