Scientists have developed an adaptable materials platform that can safely and efficiently deliver a wide range of genetic medicines, a breakthrough that could accelerate the development of next generation vaccines, cancer treatments, and genesilencing drugs.
Experts from the University of Notting- ham’s School of Pharmacy have created a new drug delivery platform that uses modular building blocks that self assemble with Ribonucleic acid — RNA — to form nanoscale delivery particles. The research has been published in Advanced Materials.
The materials incorporate a reversible ‘host-guest’ linking system, allowing the finetuning of the particles’ stability and behaviour. By simple variations in the chemical structure of the building blocks, diverse formulations can be rapidly generated suited to different therapeutic needs.
Prof Cameron Alexander, who led the research in Pharmacy, explains: “These findings demonstrate a powerful, highly tuneable system with the potential to improve the delivery of genetic medicines. By offering a flexible alternative to current delivery technologies and enabling automated, scalable manufacture, this platform could support faster development of RNAbased vaccines during future infection outbreaks, improve the effectiveness of RNA therapies in cancer, and expand treatment options for many diseases.”
The team demonstrated that RNAload- ed nanoparticles can be produced with the new materials using automated meth- ods that meet the stringent critical quality attributes required for the manufacture of RNA vaccines and therapeutics. This suggests strong potential for industrial scalability and rapid deployment.
The researchers successfully tested the materials delivering RNA into a broad range of cell types with efficiency matching or exceeding that of leading commercial transfection reagents, while showing no acute harmful effects on cells. The delivered RNAs were shown to reduce the expression of cancer-associated genes in breast tumour tissue in mice and to induce protection against H1N1 influenza in mice.
