"Design, Synthesis, and Characterization of Novel Transporters for Efficient and Safe RNA Delivery In Vitro and In Vivo"
The past decade has witnessed an explosive growth of RNA therapeutics, as evident by FDA’s approval of Onpattro (Alnylam) in 2018, Comirnaty (BioNTech/Pfizer) and Spikevax (Moderna) in 2022. The excitement toward this relatively new type of therapeutic modality has triggered many more research and development in the pipeline, spanning a variety of indications such as infectious diseases, cancer immunotherapy, genome editing, and neurodegenerative diseases. Despite their great promises, delivery of RNA has emerged as the central challenge for many of the development, as naked oligonucleotides have limited capacity to get across cellular membranes and elicit their functions in the cytosol. My PhD work aims to address this challenge by developing new generations of transporters that could deliver oligonucleotides with high efficiency, selectivity, and tolerability.