The success of messenger RNA (mRNA) vaccines to fight the SARS-CoV-2 pandemic has spurred interest in pursuing mRNA for treating other diseases like cancer, cardiovascular, metabolic, rare genetic disorders, and more. The road to development and regulatory approval of mRNA drugs can be faster than small molecules. However, challenges related to mRNA synthesis, purification, targeted delivery and immunogenicity need to be addressed before it can be widely used. Cambridge Healthtech Institute’s inaugural conference on Emerging mRNA Therapeutics highlights the efforts underway to develop mRNA as a viable drug modality for developing targeted therapies for diverse indications.