STRP

Our proposed project will develop new technology that will allow for the long-term storage of lipid nanoparticle-encapsulated RNA therapeutics in a dry rather than cold state. We have already demonstrated that RNA and lipid nanoparticles (LNPs), needed to deliver RNA into human cells, can be stabilized separately using our approach. Here, we will test our stabilization technology on RNA encapsulated within LNPs. After this proof-of-concept, we will iterate to identify optimal LNP compositions and RNA characteristics optimized for our system. Based on these experiments, and in consultation with our industry partner, Sigma-Genetics, we will down-select to a medically relevant mRNA payload that meets our performance metrics. We will then stabilize this mRNA (supplied by Sigma-Genetics) using our technology and return it to Sigma-Genetics for independent functional testing. These steps will demonstrate that our technology can preserve RNA-LNPs in the dry-state, even with industry-relevant RNA payloads. In parallel, we will conduct immunogenicity, toxicity, and preliminary pharmacokinetic studies in mice to gather critical preclinical data. Our technology directly addresses the central limitation of RNA-therapeutics – their instability outside the cold-chain. By overcoming this challenge, our technology will enable wider distribution of RNA-therapeutics to rural, developing, and underserved regions, both domestically and globally. Additionally, our technology is broadly applicable as LNPs are a platform technology meaning that their stabilization in the dry-state can be used for storage/delivery of other important biologics, such as protein-based or veterinary drugs, making it valuable across human and animal health.