STAT+: Early data from rare-disease trial put spotlight on Moderna’s efforts to turn mRNA into medicines
LONDON — Moderna may be best known for its Covid-19 vaccine, but since its start, it’s always been set on developing therapies.
It’s run into some hurdles as it’s pioneered turning mRNA — the strand of genetic material that’s at the heart of the Moderna’s approach — into medicines. But the company’s vision of making cells into their own drug factories is showing signs of progress.
On Wednesday, scientists reported interim results from an early study of Moderna’s most advanced rare-disease therapy, a treatment for propionic acidemia, a metabolic condition in which the body makes defective versions of enzymes that are required to break down fats and proteins. While the study primarily focused on safety and testing different doses, some patients — most of the participants were children — saw a reduction in the life-threatening metabolic emergencies that can crop up with the disease.
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