Compounds suited as nanocarriers for active agents and their use

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

This patent describes a new class of polyglycerol-based compounds designed to function as nanocarriers for delivering various active agents, such as genetic materials (DNA, RNA, siRNA), small molecule drugs, peptides, and metal ions, into cells. These carriers feature a biocompatible polyglycerol core with surface modifications that enable effective binding and delivery of negatively charged molecules like nucleic acids while minimizing toxicity. The innovation also includes nanocarrier systems and transfection kits using these compounds.

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

• Gene therapy for treating genetic disorders by delivering DNA, RNA, or siRNA into cells
• Cancer treatment via targeted delivery of gene-silencing molecules (e.g., siRNA for oncogene knockdown)
• Drug delivery systems for controlled transport of small molecule therapeutics or peptides into human or animal cells
• In vitro and in vivo transfection experiments in research, including for screening gene function or validating drug targets
• Delivery of metal ions or bioactive molecules for therapeutic or research purposes
• Kits for biotechnological applications such as cellular engineering, functional genomics, or therapeutic development

BenefitsContent extracted from patent full text and abstract with AI.

• High biocompatibility due to the polyglycerol core, reducing adverse reactions compared to conventional cationic delivery agents
• Lower cellular toxicity than widely used polyamines or cationic lipids, making it safer for in vivo applications
• Surface modification enables efficient binding and delivery of a broad range of negatively charged or bioactive molecules
• Customizable shell structure allows tuning of charge density and chemical functionality to optimize delivery efficiency and minimize toxicity
• Demonstrated ability to efficiently deliver siRNA and achieve high gene silencing efficacy in cell lines and animal models
• Enzymatically cleavable surface groups can improve post-delivery biocompatibility by detaching potential cytotoxic motifs inside target cells
• Versatile application across pharmaceuticals, research, and biotechnology

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

Compound suited as entity carrier, having the general formula (I) with PG denoting a linear or branched polyglycerol core, X being and being covalently bound to a carbon atom of the polyglycerol core, wherein the polyglycerol core carries a plurality of groups of the type X The invention further relates to the use of such compounds, a nanocarrier system and a kit comprising such compounds.