Infectious Diseases | Immuno-Oncology | Rare Diseases


There are approximately 7,000 rare diseases that affect more than 300 million people worldwide. An estimated 30 million people in the U.S. are living with rare diseases, or 10 percent of the U.S. population. Yet, despite this high prevalence, there are approved drugs for only 5 percent of rare diseases and disorders.

Moderna recognizes the impact of rare diseases on patients and their families, particularly with a lack of effective treatment options. We are advancing mRNA-based therapeutics with the goal of one day bringing treatment options to patients and their families.

A large number of diseases, including many rare genetic diseases, are caused by defects or deficits in proteins expressed by liver cells. By delivering mRNA therapeutics intravenously (IV), we can potentially stimulate production of therapeutic proteins in the liver in ways that cannot be achieved with other technologies. As such, rare liver diseases are our primary initial focus area.

Our first liver therapeutic development program is for methylmalonic acidemia (MMA), a serious and often life-threatening rare liver disease; it is primarily a pediatric disease with onset in early infancy.

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“As a company, we often talk about the societal responsibility we feel to deliver on the promise of mRNA science for patients. That sense of responsibility is felt with great intensity, and urgency, by the Rare Diseases team. We understand that, for so many patients suffering with a debilitating rare disease, there are no approved treatments. We are committed to leveraging our mRNA platform to advance medicines for some of these diseases and bring new hope to patients and their families.”

- Paolo Martini, Chief Scientific Officer, Rare Diseases