Ionizable cationic lipids with conserved spacing and lipid nanoparticles
Ionizable cationic lipids, such as CICL-208 to CICL-235, are used to formulate LNPs and tLNPs, addressing issues of off-target delivery and payload release efficiency, while reducing toxicity associated with lipid accumulation.
WO2025076113A1 Publication Date: 2025-04-10CAPSTAN THERAPEUTICS INC
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Patent Information
- Application Number
- PCT/US2024/049627
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-05-31
- Filing Date
- 2024-10-02
- Publication Date
- 2025-04-10
AI Technical Summary
Technical Problem
Current lipid formulations for delivering nucleic acids into cells face challenges such as off-target delivery, poor efficiency of releasing therapeutic agents into the cytoplasm, and toxicity associated with lipid accumulation.
Method used
Development of ionizable cationic lipids with specific structures, such as CICL-208, CICL-227, CICL-228, CICL-229, CICL-233, CICL-234, and CICL-235, which are used to formulate lipid nanoparticles (LNPs) and targeted LNPs (tLNPs) for enhanced delivery efficiency.
Benefits of technology
The use of these ionizable cationic lipids in LNPs and tLNPs improves the targeted delivery of nucleic acids, increases the efficiency of payload release into the cytoplasm, and reduces toxicity by minimizing lipid accumulation.
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Abstract
Ionizable cationic lipids, methods for synthesizing the same, intermediates useful in synthesis of the ionizable cationic lipids, and methods of synthesizing the intermediates are disclosed. The ionizable cationic lipids are useful as a component of lipid nanoparticles (LNP), which in turn can be used for delivering nucleic acids into cells in vivo or ex vivo. LNP compositions are also disclosed, including LNP comprising a functionalized lipid to enable conjugation of a binding moiety, and targeted LNP (tLNP), that is a LNP in which a binding moiety has been conjugated to the functionalized lipid and can serve as a targeting moiety to direct the tLNP to a desired tissue or cell type.
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