Nature Reviews Drug Discovery, Published online: 03 June 2026; doi:10.1038/s41573-026-01459-7 mRNA technology is being applied to develop a new generation of therapies for rare and common diseases. This Review discusses insights from clinical studies on mRNA-based therapeutics, including enzyme replacement therapies, cancer immunotherapies, genome-modifying therapies and immune cell reprogramming therapies, which illustrate how technological advances are paving the way for the wider adoption of