In first look at mRNA protein replacement, Moderna touts early signals of promise for rare metabolic therapy
LOS ANGELES — Moderna presented early data on its mRNA treatment for propionic acidemia (PA), a rare metabolic disease where people can’t break down certain proteins and fats, suggesting patients had fewer severe attacks characteristic of the disease following treatment.
It’s the first time Moderna has shared clinical results of an mRNA treatment designed to replace broken or missing proteins inside a cell. If the technique proves safe and effective in larger studies, it could unlock a swathe or new applications for mRNA.
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