On a brisk morning in April, dozens of investors filed into a Marriott hotel conference hall in Kendall Square, the biotech hub of Cambridge, Mass., for the first Moderna Therapeutics platform science day. They gathered hoping to get a rare, inside look at the science behind what the start-up had long claimed to be a disruptive drug platform: carefully designed molecules called messenger RNA that prompt the body to make its own medicine. The concept has attracted billions in funding, but the company had largely kept details of how the technique works under wraps.
“Why are we so passionate about messenger RNA?” Moderna President Stephen Hoge asked the attentive audience. “It starts with the question of life,” he explained. “And in fact, all life that we know flows through messenger RNA. … In our language, mRNA is the software of life.”
Cells use mRNA to translate the static genes of DNA into dynamic proteins, involved in every bodily function, Hoge explained. Biotech companies make some of these proteins as drugs in large vats of genetically engineered cells. It’s a time-consuming and costly process.
Moderna offered a different proposition: What if instead, mRNA was given therapeutically? In theory, it could prompt proteins to be made in your body. It would put the drug factory inside you.