Ligands for extrahepatic delivery
Oligonucleotides with lipophilic moieties at the 5' and 3' ends enhance siRNA delivery to extrahepatic tissues, addressing the limitations of current therapies and expanding their applicability beyond liver-related diseases.
AU2025205687A1Pending Publication Date: 2026-07-23RONA BIOSCIENCE LTD
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Patent Information
- Application Number
- AU2025205687
- Authority / Receiving Office
- AU · AU
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-08-09
- Filing Date
- 2025-01-03
- Publication Date
- 2026-07-23
AI Technical Summary
Technical Problem
Current siRNA therapies primarily focus on liver-related diseases and face challenges in delivering siRNA to extrahepatic tissues such as the eyes or central nervous system, limiting their application.
Method used
Development of oligonucleotides with lipophilic moieties at the 5' and 3' ends, enhancing extrahepatic delivery by incorporating compounds of formula (I) to improve targeting and efficacy.
Benefits of technology
The proposed solution enables more efficient extrahepatic delivery of siRNA, overcoming limitations in current therapies and expanding their applicability to non-liver tissues.
✦ Generated by Eureka AI based on patent content.
Abstract
The present invention provides an oligonucleotide, wherein the oligonucleotide has at least 15 nucleotides sufficiently complementary to the target mRNA, wherein one or more sites of the oligonucleotide are conjugated to a lipophilic moiety.
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