Lipid nanoparticles for delivery of therapeutic payloads to cells

By employing novel ionizable lipid formulations and targeting moieties, the lipid nanoparticles address the challenges of payload delivery, toxicity, and cell specificity, achieving significantly improved transfection efficiency and expression in target cells.

WO2025059596A1 Publication Date: 2025-03-20TESSERA THERAPEUTICS INC
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Patent Information

Application Number
PCT/US2024/046800
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-09-13
Filing Date
2024-09-13
Publication Date
2025-03-20

AI Technical Summary

Technical Problem

Current lipid nanoparticles (LNPs) face challenges in delivering sufficient payloads to target cells, particularly for larger payloads or multiple payloads like those used in gene editing. Additionally, LNPs often exhibit unfavorable toxicological profiles and struggle with cell-type specificity, transfection efficiency, and endosomal escape.

Method used

The development of lipid nanoparticles (LNPs) with a novel ionizable lipid formulation, which includes specific lipid structures and compositions, such as Lipid 093, Lipid 176, Lipid 178, Lipid 183, and Lipid 235, to enhance payload delivery to cells like T cells and hematopoietic stem cells (HSCs). These LNPs may include a targeting moiety for improved cell specificity and are designed to facilitate higher transfection efficiency and endosomal escape.

Benefits of technology

The proposed LNPs demonstrate enhanced delivery efficiency, achieving higher transfection rates and payload expression in target cells compared to baseline LNPs. This is reflected in increased transduction and expression levels in both animal models and humanized systems, with specific formulations showing up to 99% more efficient delivery to certain cell types.

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Abstract

The disclosure provides ionizable lipids for constructing lipid nanoparticles. The disclosure further provides conjugates comprising a targeting moiety a lipid nanoparticle (LNP) encapsulating a therapeutic agent (i.e., payload) for delivery to immune cells or hematopoietic stem cells (HSCs). The conjugates can be formulated in a pharmaceutical composition and can be directly administered to a patient in need of (i.e. in vivo administration).
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