Moderna's mRNA therapy, mRNA-3927, offers potential treatment for the rare genetic disorder propionic acidemia, affecting 1 in 100,000 in the U.S.

The therapy works by delivering mRNA to produce enzymes that are deficient in those with the disorder.

In a recent trial involving 16 patients, mRNA-3927 led to a 70% reduction in metabolic decompensation events, a serious complication of the disease.

The treatment's side effects were present but not severe enough to stop or reduce dosages.

Published research in April 2024 indicates progress in treating propionic acidemia and methylmalonic acidemia, with the trial ongoing to determine long-term safety and efficacy.

Further research is required to confirm mRNA-3927 as a long-term solution for genetic diseases.