Nucleic acids for inhibiting expression of GPR146 in a cell
Double-stranded nucleic acids with precise sequences target GPR146 expression to treat hypercholesterolemia and atherosclerosis, addressing off-target issues and hepatotoxicity in RNA interference therapies.
AU2025214336A1Pending Publication Date: 2026-07-23SILENCE THERAPEUTICS GMBH
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Patent Information
- Application Number
- AU2025214336
- Authority / Receiving Office
- AU · AU
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-01-29
- Filing Date
- 2025-01-28
- Publication Date
- 2026-07-23
AI Technical Summary
Technical Problem
Existing RNA interference-based therapies for GPR146-mediated diseases suffer from off-target effects and hepatotoxicity, limiting their efficacy and safety for treating conditions like hypercholesterolemia and atherosclerosis.
Method used
Development of double-stranded nucleic acids with specific sequences that differ by no more than 3 nucleotides from reference sequences, designed to inhibit GPR146 expression with minimal off-target effects, formulated in compositions for therapeutic and diagnostic use.
Benefits of technology
The nucleic acids effectively reduce GPR146 expression with reduced off-target effects, providing a safer and more effective treatment for hypercholesterolemia, hypercholesterolaemia, atherosclerosis, and related cardiovascular diseases.
✦ Generated by Eureka AI based on patent content.
Abstract
The invention relates to double-stranded nucleic acid molecules that interfere with or inhibit G Protein-Coupled Receptor 146 (GPR146) gene expression. It further relates to therapeutic uses of such inhibition such as for the treatment of GPR146-mediated diseases, disorders or syndromes, such as hyperlipidaemia, hypercholesterolaemia, Heterozygous familial hypercholesterolaemia (HeFH), Homozygous familial hypercholesterolaemia (HoFH), autosomal recessive hypercholesterolaemia, atherosclerosis, and / or atherosclerotic cardiovascular disease.
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