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 gene‑silencing drugs.
Experts from the University of Nottingham'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 fine‑tuning 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.
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 RNA based vaccines during future infection outbreaks, improve the effectiveness of RNA therapies in cancer, and expand treatment options for many diseases.
The team demonstrated that RNA‑loaded nanoparticles can be produced with the new materials using automated methods 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.
This work involved a multidisciplinary team spanning the University of Nottingham, Imperial College London, King's College London, University College London and two Cambridge-based spinouts, Aqdot Ltd and Centillion Ltd.