Delivering Large Gene Editing Tools into Cells with Hybrid Virus-Lipid Packages

This patent describes a sophisticated delivery system that combines parts of a virus with a fat bubble (liposome) to efficiently transport large molecules, like CRISPR gene-editing components, into specific cells.

What it covers

This patent describes a "particle delivery system" (Claim 1) designed to get large molecules into cells. It uses a "composite virus particle" which is a tiny structure made of a specific lipid, a piece of a virus's outer shell (a "virus capsid protein"), and another large protein or peptide that isn't part of the virus shell (a "non-capsid protein or peptide"). This composite particle is then attached to a "liposome" (a small fat bubble) that has a "targeting moiety" — a special tag that helps it find and attach to specific cells (Claim 1). The system can carry a "CRISPR system component" (Claim 3) or other large proteins up to a megadalton in size (Claim 9). For example, this system could be used to deliver the Cas9 protein, a key part of CRISPR gene editing, into a specific type of cell in the body, like a liver cell, by using a targeting moiety that recognizes that cell type.

What it doesn't cover

• —Does not cover delivery systems that use only a virus or only a liposome, as it requires a "composite virus particle" adsorbed to a "liposome" (Claim 1).
• —Does not cover systems using lipids outside the specific list provided in Claim 1 (e.g., EC16-63, 80-O14B, etc.).
• —Does not cover liposomes without a "targeting moiety," which is a specific component for guiding the delivery (Claim 1).
• —Does not cover delivery of very small molecules or payloads that are not proteins or peptides, as the "non-capsid protein or peptide" is described as having a molecular weight up to a megadalton and specifically mentions CRISPR proteins (Claim 9).
• —Does not cover attachment methods between the composite virus particle and the liposome that are not hydrophobic or electrostatic interactions (Claim 2).

The clever bit

The clever part is combining the best features of viral delivery and lipid nanoparticle delivery into one system. It uses a "composite virus particle" that is then attached to a "liposome" with a "targeting moiety," creating a highly specific and efficient way to get large, sensitive payloads like CRISPR components into cells while potentially reducing unwanted immune responses.

Why it matters

This technology is crucial for advancing gene therapy and genetic medicine. Efficient and safe delivery of large therapeutic molecules, especially gene-editing tools like CRISPR, into target cells remains a major challenge. By combining the cell-entry efficiency of viruses with the targeting and safety benefits of liposomes, this patent offers a pathway to overcome some of these limitations, potentially enabling new treatments for genetic diseases.

Real-world examples

• 1.Experimental gene therapies using CRISPR-Cas9
• 2.Advanced drug delivery systems for biologics
• 3.Targeted delivery of mRNA vaccines or therapeutics