In DepthDRUG DEVELOPMENT

mRNA's next challenge: Will it work as a drug?

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Science  18 Dec 2020:
Vol. 370, Issue 6523, pp. 1388-1389
DOI: 10.1126/science.370.6523.1388

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Summary

The dramatic success of two COVID-19 vaccines in clinical trials last month marked a triumph for a previously unproven medical technology. The vaccines, one of which was authorized for emergency use by the U.S. Food and Drug Administration last week, rely on the genetic instruction known as messenger RNA (mRNA). It prompts cells to make a SARS-CoV-2 protein that trains the immune system to recognize the virus. But long before the pandemic, mRNA tantalized pharma, promising a simple and flexible way to deliver both vaccines and drugs. Now, the vaccine wins have created new enthusiasm around the concept. But mRNA medicines—especially those that replace beneficial proteins for chronic disease—have a tougher road to the clinic than vaccines.

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