Compositions of Polyplexes and Saponins

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

This invention relates to advanced non-viral methods for delivering nucleic acids (such as therapeutic DNA or RNA) into target cells, using specially designed polyplex compositions. It describes a system where nucleic acids are complexed with a polymeric scaffold and combined with saponins (a type of natural compound) that enhance the escape of the nucleic acids from endosomes inside the cells. The saponin is either covalently attached to the polymeric scaffold or to a cell-targeting ligand, via a special linker that only releases the saponin under conditions present in endosomes. These compositions can be tailored for targeted and efficient gene delivery for therapeutic purposes.

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

• Systemic, targeted gene therapy for treating genetic disorders (e.g., hemophilia B) by delivering therapeutic DNA to specific organs like the liver.
• Treatment of cancers by delivering toxin-encoding nucleic acids or gene-editing tools specifically to cancer cells (e.g., using ligands targeting EGFR or Her2 receptors).
• Non-viral delivery of gene-editing components (e.g., CRISPR-Cas9) for in vivo genome editing.
• Delivery of RNA interference (RNAi) therapies to modulate gene expression in diseased cells.
• Ex vivo modification of cells (e.g., adoptive cell therapies) using non-viral gene delivery before re-infusion into patients.
• Potential application in vaccine development by efficiently delivering RNA/DNA encoding antigens to immune cells.

BenefitsContent extracted from patent full text and abstract with AI.

• Overcomes the main limitation of poor endosomal escape and cytosolic delivery faced by current non-viral gene therapies.
• Reduces or eliminates the use of viral vectors, thereby minimizing associated safety concerns such as immune reactions and high manufacturing complexity.
• Highly modular: allows for precise targeting to specific cell types or tissues through conjugation with targeting ligands (e.g., antibodies, GalNAc).
• Increases efficiency and specificity of gene delivery, potentially leading to lower therapeutic doses and reduced side effects.
• Supports the delivery of a wide range of nucleic acids, including large plasmids, mRNA, siRNA, and gene-editing constructs.
• Demonstrates stability in blood and low toxicity in vitro and in vivo, making it suitable for systemic administration.
• Can be cost-effective and scalable for broader patient access compared to viral-based methods.
• Adaptable for personalized medicine applications, including precision gene editing.

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 is a nucleic acid that is polyplexed with a polymeric scaffold, which is provided in combination with an endosomal escape-enhancing saponin that is covalently bound either to the polymeric scaffold or to a cell targeting ligand by a linker configured to release the saponin from the scaffold under conditions present in an endosome. The disclosed herein compositions and methods may be exploited in the treatment of various diseases and/or conditions by systemic delivery.