Session 4: mRNA and Self-Amplifying RNA (saRNA): Opportunities for Disease Prevention and Therapy

Toxicol Pathol. 2024 Nov 22:1926233241298572. doi: 10.1177/01926233241298572. Online ahead of print.

Abstract

The unprecedented speed of developing vaccines against the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), responsible for the COVID-19 pandemic, has propelled mRNA technologies into the public eye. The versatility of mRNA technology, often referred to as "plug and play," offers immense promise for rapidly updating vaccines to address newer variants of respiratory diseases and combat emerging infectious diseases and lethal pathogens, such as the Ebolavirus. However, the potential applications of mRNA technology extend well beyond prophylactic vaccines. This session explored the two primary mRNA platforms: nonreplicating mRNA and self-amplifying mRNA (variably referred to as saRNA, samRNA, or SAM). Presentation topics were on current research efforts aimed at broadening the applications of mRNA modalities beyond vaccines. Topics included opportunities for delivering mRNA via intra-tumoral and inhalational routes, immunological and systemic inflammatory responses elicited by these modalities, and regulatory considerations involved in the development and licensing of these technologies.

Keywords: RNA therapeutics; mRNA; nonclinical; pathology; regulatory; saRNA; safety; samRNA; self-amplifying mRNA; vaccines.