Methods and Compositions for Enhanced Polyplex Delivery

Simple SummaryContent extracted from patent full text and abstract with AI.

This invention provides a new method and composition for enhancing the delivery of nucleic acids (like DNA or RNA) into specific target cells without using viral vectors. The patented technology combines two components: (1) a polyplex (a complex of nucleic acid and a polymer) and (2) a saponin (a plant-derived molecule) linked to a cell-targeting ligand. The saponin helps the nucleic acid escape from the endosomes inside the cell, ensuring the genetic material can reach the cytosol and function effectively, but only in the cells that are specifically targeted by the ligand. This results in targeted, safer, and more efficient gene delivery for therapeutic uses.

Use CasesContent extracted from patent full text and abstract with AI.

• Targeted gene therapy for diseases such as cancer, genetic disorders, and rare diseases without viral vectors.
• Delivery of gene-editing tools like CRISPR/Cas9 specifically to diseased cells (e.g., tumor cells) in vivo.
• Personalized medicine applications, where genetic material can be delivered precisely to patient-specific cell types.
• Ex vivo gene modification of cells (such as immune cells) for cell-based therapies, then re-introduction into patients.
• Delivery of large DNA constructs (such as plasmids or therapeutic genes) in a more stable, safe, and cell-specific manner.

BenefitsContent extracted from patent full text and abstract with AI.

• Highly targeted delivery of nucleic acids, minimizing off-target effects and toxicity to non-diseased cells.
• Avoids the risks and manufacturing complexities associated with viral vectors (e.g., immune reactions, high cost).
• Facilitates efficient endosomal escape, addressing a major bottleneck in non-viral gene delivery systems.
• Can be used for larger genetic payloads than many competing delivery methods.
• Compositions show stability in blood and low toxicity, indicating suitability for systemic (whole-body) administration.
• Modular and adaptable – choice of ligand allows targeting to different cell types or disease states.
• Potentially lower production costs, supporting broader patient access to advanced gene therapies.

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

The invention lies in the field of delivery of nucleic acids into a cell. In particular, disclosed herein are compositions comprising at least two components being (i) a polyplex comprising a nucleic acid to be delivered into a cell; and (ii) a covalent conjugate of an endosomal-escape-enhancing saponin bound to a cell targeting ligand by a linker configured to release the saponin from the cell targeting ligand under conditions present in an endosome. Thanks to the combination of the at least two components (i) and (ii), the presented herein compositions and methods achieve enhanced cytosolic release of the nucleic acid specifically in the cells targeted by the cell targeting ligand bound to the saponin.