mRNA Technology Beyond Vaccines: Exploring Potential mRNA-Based Therapies for Autoimmune Diseases

Messenger RNA (mRNA) technology, which has attracted considerable attention because of new vaccines for COVID-19, is now being explored for new treatment possibilities outside of infectious disease. One of the most tantalizing uses of mRNA technology is mRNA therapeutics for treating and modulating autoimmune disease. Autoimmune diseases, such as multiple sclerosis, rheumatoid arthritis, and type 1 diabetes, occur because of aberrant immune immune responses that attack self-tissue. Standard treatment for autoimmune disease is generally a form of broad immunosuppression that can have unwanted side effects and increased risk of infection. mRNA technology offers a more target and potentially safer way to direct cells to produce therapeutic proteins that promote immune tolerance or modulate inflammatory pathways. Recent preclinical and early clinical studies demonstrate that mRNA constructs are capable of selectively modulating immune responses, either by encoding tolerogenic antigens or cytokine regulators, or by submitting cell specific peptides. The mRNA platform also allows for rapid customization and scaling of therapies, which represents a significant opportunity for developing personalized therapies built around each patient’s specific immunologic profile. There are still hurdles related to improving delivery systems, sustaining durability of response, and limiting off-target effects, however, enhancements in lipid nanoparticle formulations and in the engineering of mRNA sequences are quickly resolving these issues. As the field continues to advance, mRNA targeted therapies can potentially revolutionize care of autoimmune diseases and give hope for prolonged periods of remission with improved quality of life outcomes. This review will summarize the current state of the art, recent advances, and future potential of mRNA therapeutics in the care of autoimmune disease.