Sirna molecule for regulating PCSK9 gene activity
A novel siRNA with modified nucleotides and a targeted ligand moiety addresses stability and absorption issues, effectively inhibiting PCSK9 expression to treat hyperlipidemia and atherosclerosis.
AU2023295621B2Pending Publication Date: 2026-07-23RONA BIOSCIENCE LTD
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Patent Information
- Application Number
- AU2023295621
- Authority / Receiving Office
- AU · AU
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2022-06-14
- Filing Date
- 2023-06-13
- Publication Date
- 2026-07-23
AI Technical Summary
Technical Problem
Current siRNA therapies for inhibiting PCSK9 expression face challenges such as poor stability, rapid degradation by nucleases, difficulty in tissue absorption, and off-target effects, limiting their clinical application in treating hyperlipidemia and atherosclerosis.
Method used
Development of a novel small interfering RNA (siRNA) with specific sense and antisense strands, modified nucleotides, and a conjugated ligand moiety to enhance stability and targeting, specifically designed to inhibit PCSK9 expression in cells.
Benefits of technology
The siRNA effectively reduces PCSK9 expression, potentially treating conditions like hyperlipidemia, atherosclerosis, and other diseases by enhancing cellular uptake and minimizing off-target effects.
✦ Generated by Eureka AI based on patent content.
Abstract
Provided are a small interfering RNA (siRNA) used to inhibit the expression of proprotein convertase subtilisin / kexin type 9 (PCSK9) in a cell, a vector comprising nucleotides encoding same, and a cell, as well as a method using the siRNA, the vector or the cell for the treatment of diseases or symptoms associated with the expression of PCSK9 in a subject.
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