Messenger RNA (mRNA) plays a critical role in human biology, providing a set of instructions to cells in the body to make proteins. Moderna is developing an investigational mRNA medicine (mRNA-3704) for methylmalonic acidemia (MMA) that provides instructions to cells in the liver to make normal MUT protein. It is hoped that by increasing the MUT proteins, the buildup of harmful substances in the liver and blood that are causing symptoms can be reduced.
 
A Phase 1/2 study will evaluate the safety of mRNA-3704 is as well as its effectiveness in children 1-18 years of age with MMA who have not received a liver or kidney transplant. Up to 34 children with MMA are expected to take part in the study in multiple research institutions throughout the United States. The first of these sites recently opened. You can read more about the trial here.
 
Moderna is a biotechnology company focused on developing a new class of medicines using mRNA. Moderna is developing therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and cardiovascular diseases, independently and with strategic collaborators. As of May 2019, the Company has 21 development candidates in its pipeline, with 11 in clinical trials.
 
MMA is a genetic disease that is primarily caused by an enzyme named methylmalonyl CoA mutase (MUT) that does not work properly.  MUT helps to break down foods that we eat.  When MUT does not work, this causes a buildup of other substances (such as methylmalonic acid) in the body that can be harmful. 
 
To learn more about this Phase 1/2 study, please contact your doctor, visit the clinical trial summary online or email clinicaltrials@modernatx.com. 
 
To learn more about Moderna, please visit modernatx.com.