glycan-functionalized lipid nanoparticles for targeted mrna delivery

RNA therapeutics have emerged as a revolutionary approach to treating diseases, offering a powerful platform for delivering therapeutic RNA molecules encapsulated in lipid nanoparticles (LNPs). These LNPs protect the RNA in the bloodstream, enhancing its stability and ensuring it reaches its target cells. The recent success of mRNA-based COVID-19 vaccines has demonstrated the immense potential of RNA therapeutics, showing their capacity to respond quickly to emerging health threats and provide effective treatments for previously difficult-to-treat diseases. Despite these advancements, a significant challenge remains: delivering these therapies efficiently and precisely to the correct cells to maximize therapeutic benefit while minimizing off-target effects. This project aims to address this challenge by using glycoscience through the application of glycan-targeted LNPs, a novel approach that leverages specially designed sugar molecules (glycans) to direct the LNPs to specific cell types. Glycans naturally interact with proteins on the surfaces of cells, making them ideal for targeting immune cells or inflamed endothelial cells, both of which play critical roles in immune responses and disease progression. The collaboration between NeoVac, a leader in LNP technology, and Sussex Research Laboratories (SRL), a leader in glycan chemistry and the development of glycan targeting ligands, will drive the development of LNPs that can deliver RNA therapies with greater precision. Our project will focus on synthesizing and screening various glycan ligands that bind to target cells. These ligands will then be incorporated into LNPs and evaluated for their ability to effectively deliver RNA to specific cell types. Successful outcomes will be validated using disease models to ensure therapeutic relevance. The development of glycan-targeted LNPs has the potential to transform RNA therapy delivery, enhancing precision and reducing the risk of side effects, the result of commonly encountered off-target effects. This project could pave the way for new therapeutic approaches to diseases such as cancer, autoimmune disorders, and infectious diseases. Ultimately, the goal is to create a scalable and adaptable platform for targeted RNA delivery, offering a safer and more efficient method for treating complex diseases. If successful, this technology could significantly advance the field of precision medicine and improve outcomes in RNA-based therapies.