“Designed by Moderna in Cambridge, Massachusetts, the current therapy uses mRNA technology to restore metabolic function in people with propionic acidaemia.

This rare genetic disorder, which affects about one in 100,000 individuals worldwide, arises from mutations in either of two genes that together encode an enzyme necessary for the efficient breakdown of certain protein components. Without this enzyme, cells can’t process some nutrients properly.

That leads to the accumulation of toxic chemicals in the blood and tissues, and damages vital organs, including the heart and the brain. Symptoms, such as vomiting, usually start within the first few days after birth.”

From Nature.