We are developing better ways to deliver genetic medicines to the heart. These treatments use small pieces of RNA to help repair heart tissue but targeting them into heart cells is difficult.
We are testing fat-based particles called lipid nanoparticles (LNPs) to carry RNA safely and effectively to the heart. By comparing different LNP designs in our in vivo models, we aim to find the best approach to deliver future therapies to the heart.
Cardiac deliveryIn depth
One of the bottlenecks for any application involving nucleic acid administration to the heart is efficiency of cardiac delivery. In the cases of both coding mRNA or RNA interference therapeutics (miRNAs, siRNAs), delivery needs to be facilitated in order for these molecules to cross the plasma membrane, whether of cardiomyocytes, fibroblasts or endothelial cells.
We aim to evaluate the efficiency of lipid nanoparticles (LNPs) produced using SNALP technology for cardiac gene transfer. Our objective is to systematically compare LNP formulations incorporating diverse ionizable lipids, lipidoids, and PEGylated lipids, alongside lipids sourced both from academic and commercial collaborators. This comparison will include optimisation of lipid composition and assessment of delivery routes across two in vivo models, focusing on transfection efficiency and distribution within cardiac cell populations.