Rare Disease
at Moderna

About mRNA

in Rare Diseases

Moderna is committed to researching and developing mRNA therapies for patients living with rare diseases. There are approximately 7,000 rare diseases that affect more than 300 million people worldwide.1 Yet there are approved drugs for only 5 percent of rare diseases.2 We understand the urgency with which patients and caregivers are waiting for potentially novel therapeutic options, so we are working tirelessly to research potential mRNA treatments.

mRNA is a messenger molecule that carries instructions for making proteins to cells. This science could be applied to diseases where the body does not properly make protein, like many rare diseases.

1 https://www.rarediseasesinternational.org/new-scientific-paper-confirms-300-million-people-living-with-a-rare-disease-worldwide

2 https://rarediseases.org/wp-content/uploads/2019/01/RDD-FAQ-2019.pdf

Kyle Holen, MD, Head of Development, Therapeutics, and Oncology image

Rare Disease Research

Glycogen Storage Disease Type 1a (GSD1a)

GSD1a (also known as von Gierke disease) is a disease where the body has a buildup of glycogen, a complex sugar. This buildup prevents organs and tissue from functioning properly.

Propionic Acidemia (PA)

People who are diagnosed with propionic acidemia (PA) are missing the propionyl-CoA carboxylase (PCC) enzyme, which is responsible for the breakdown of certain proteins and fat. When this enzyme is missing, it is difficult for cells in the body to turn food into energy, leading to toxins building up in the bloodstream.

Moderna’s Rare Disease Trials


A Clinical Trial of a Glycogen Storage Disease Type 1a (GSD1a) Treatment for Adults with mRNA-3745


A Clinical Trial of a Treatment for Patients with Propionic Acidemia


A Clinical Trial of a Treatment for Patients with Methylmalonic Acidemia (MMA)


To learn more about any of our rare disease clinical trials or to see if you may qualify to join, please contact the Moderna Clinical Trial Support Center.

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Discover new treatments. Advance medical research. Potentially gain access to investigational treatments.

At Moderna, we are dedicated to creating and developing innovative mRNA vaccines and therapies for a wide range of diseases that impact people’s lives, but we can’t do it alone. We invite you to partner with us on our journey to bring a new generation of transformative medicines to the world. Only people can help save people.


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