A rare disease is any disease for which the number of affected patients is below a certain level. In U.S., it is less than 200,000 patients per disease, and in South Korea it is less than 20,000 patients per disease (based on U.S. FDA and Korea FDA information). According to the WHO, nearly 7,000 rare diseases have been reported so far and 3.5 - 5.9% of the worldwide population are suffering from one or more rare disorders. There are about 700 drugs approved by the U.S. FDA and it covers only 5% of entire rare diseases, leaving huge unmet medical needs in the field.

Rare diseases are generally caused by genetic defects, thus its pathophysiology is with dysfunctional proteins. An emerging mRNA technology can produce proteins of interest efficiently when delivered to the body by using the host as a bioreactor. MOGAM believes that mRNA medicine will open the new door for protein replacement therapy and it will meet the unmet medical needs of numerous rare disorders.

The symptoms of rare diseases are biologically complex to understand and it can be challenging to uncover its genomic or pathological drivers. Replacing the missing protein using mRNA technology is the first step we take for a new treatment. And our scientific dedication to understand the complexity of disease conditions will further advance the treatment.

MOGAM hopes that mRNA therapy will ease the physical, financial and psychological burdens on patients with rare diseases and their families.