Moderna’s mRNA Platform for Rare Diseases

Ruchira Glaser, MD, MS, Senior Vice President, Therapeutic Area Head of Rare Disease, Autoimmune & Cardiovascular at Moderna, discusses the company’s leading mRNA platform.
As Dr. Glaser notes, the use of mRNA therapies is not novel; however, Moderna’s platform has demonstrated new ways to use mRNA therapies to prevent and treat diseases, particularly those that are rare.
Dr. Glaser goes on to discuss how mRNA therapies work. As she explains, all mRNA therapies begin with the identification of a protein that can either prevent or treat a disease. mRNA therapy is then engineered to instruct cells in the body to make that specific protein.

Moderna’s mRNA platform is investigating mRNA therapy in a wide range of diseases from rare metabolic diseases, like methylmalonic acidemia (MMA) and propionic acidemia (PA), to cancers, as well as cardiovascular diseases.