Ionizable lipids for lipid nanoparticles
By combining ionizable lipids of formula (I) with other lipid components to form lipid nanoparticles, the problem of lipid nanoparticle aggregation in solution is solved, improving the stability and delivery efficiency of mRNA therapeutics and making them suitable for various routes of administration, especially local delivery in cystic fibrosis.
Patent Information
- Authority / Receiving Office
- CN Β· China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- MERCK PATENT GMBH
- Filing Date
- 2024-10-15
- Publication Date
- 2026-05-26
AI Technical Summary
Existing lipid nanoparticles rapidly aggregate in solution, leading to reduced activity of mRNA therapeutics at standard refrigerator temperatures, which limits their home storage and clinical translation, particularly for local delivery of chronic diseases such as cystic fibrosis. Furthermore, existing delivery systems are inadequate in protecting nucleic acids from degradation, cell entry, and endosome escape.
The ionizable lipids of formula (I) are combined with neutral lipids, cholesterol and polyethylene glycol-modified lipids to form lipid nanoparticles, which optimize the nucleic acid/lipid ratio, enhance cellular uptake and protect nucleic acids, reduce toxicity, and are easily eliminated in vivo, making them suitable for systemic and local delivery.
It improves the stability and delivery efficiency of lipid nanoparticles, reduces toxicity, ensures effective intracellular delivery of nucleic acids and long-term storage stability, and is suitable for intravenous, intramuscular, subcutaneous or intratumoral administration.
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