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A first-of-its-kind mRNA drug falls short in cystic fibrosis

Messenger RNA technology, which uses synthetic, genetic material to teach the body to make helpful proteins, has come of age over the past year.

When the coronavirus pandemic took hold in 2020, the technology was uniquely suited to meet the moment, having already shown potential as a new way to quickly design vaccines. The ensuing success of two similar mRNA vaccines from Moderna as well as partners BioNTech and Pfizer validated the technology, and the shots have since been used to protect millions of people around the world from COVID-19.

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