Nanoscale delivery platforms for RNA therapeutics: Challenges and the current state of the art

RNA nanomedicines present a promising class of therapeutics, with broad applications in protein replacement therapy, gene editing, immunotherapy, and vaccines, owing to their versatility and precise nature. Although recent years have seen dramatic improvements in the safety and efficacy of RNA-based therapies, their functional delivery to target tissues and cells in vivo remains challenging. Here, we discuss many of these challenges, as well as the methods and materials that have been developed to overcome them, with a focus on polymeric and lipid-based nanoscale delivery systems. In addition, we provide an overview of recent clinical and pre-clinical developments in RNA nanomedicines that have been made possible by advances in delivery. RNA nanomedicines present a promising class of therapeutics, with broad applications in protein replacement therapy, gene editing, immunotherapy, and vaccines, owing to their versatility and precise nature. Although recent years have seen dramatic improvements in the safety and efficacy of RNA-based therapies, their functional delivery to target tissues and cells in vivo remains challenging. Here, we discuss many of these challenges, as well as the methods and materials that have been developed to overcome them, with a focus on polymeric and lipid-based nanoscale delivery systems. In addition, we provide an overview of recent clinical and pre-clinical developments in RNA nanomedicines that have been made possible by advances in delivery.