Elpidera is a Moderna venture focused on advancing mRNA-based therapeutics for the treatment of 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. It is for this reason that we chose the name Elpidera for our rare disease-focused venture; it comes from the Greek word Elpida, meaning “hope.”
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 genetic diseases of the liver are the primary initial focus of the discovery programs Elpidera is pursuing.
“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 Elpidera 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 for Elpidera