The Future of RNA Therapeutics, From Design to Delivery
The COVID-19 pandemic catalyzed renewed interest in RNA therapeutics, which hold promise for drugging the undruggable.
RNA therapeutics have existed since the late 90s but have only come into focus in the last few years. This renewed interest has largely been attributed to the success of messenger RNA (mRNA) vaccines employed during the COVID-19 pandemic. These vaccines helped beat back the virus and showed that mRNA was safe, efficacious and could work at scale.
Following the success of mRNA vaccines, there has been a surge in interest in RNA therapies, which saw a 53% increase in trial initiations in the last quarter of 2024. In contrast to conventional protein-targeting and DNA-based medicines, RNA can theoretically target any gene of interest. This opens the possibility of treating rare conditions and targeting previously “undruggable” proteins. In this article, we will explore the current landscape of RNA therapeutics and discuss some of the innovative technologies designed to realize the full potential of these therapies.