Moderna Snags $25M DARPA Grant to Fight Pandemics With mRNA Drugs

10/2/13Follow @xconomy

One of the wilder ideas in biotechnology just got a vote of confidence from the people who gave the world the Internet.

Cambridge, MA-based Moderna Therapeutics is announcing later today it has snagged a grant worth as much as $24.6 million over the next five years from the Defense Advanced Research Projects Agency (DARPA). The U.S. military’s futuristic technology agency is committing the cash now to further develop Moderna’s messenger RNA drug technology to fight infectious diseases.

By making this grant, the agency is betting on what could become a superfast, cheap, and unusually adaptable method for fighting today’s known pandemic threats, and the unknown threats of the future.

The deal is the latest coup for Moderna, a three-year-old startup that turned heads in the biotech industry in March when it secured a huge $240 million upfront payment from AstraZeneca (NYSE: AZN) to collaborate on 40 different mRNA drug candidates. Moderna, which has grown to 80 employees and aspires to be the first mover in the field of injectable messenger RNA drugs, hasn’t yet taken any drug candidates into clinical trials. But it has quietly been doing work behind the scenes on a small $700,000 DARPA grant this year to prove that its mRNA technology is more than just science fiction.

After 18 months of talks, scientists at Moderna showed the agency that its modified mRNA molecules, which carry the instructions for making specific proteins, could get inside cells of live animals. Not only that, but once there, the mRNA could coax the cellular machinery to churn out therapeutic-quality antibodies like Genentech’s trastuzumab (Herceptin), and make more than enough of the antibody to have a therapeutic effect. That was enough for DARPA to double down and ask whether Moderna can make more mRNAs that can help people produce all kinds of other antibodies, particularly ones of national security interest.

Scientifically, it means Moderna has shown animals can serve as their own drugmaking factories. Next step: carefully marching ahead to prove it can do the same thing in human beings.

Stephane Bancel, CEO of Moderna Therapeutics

Stephane Bancel, CEO of Moderna Therapeutics

“This is a big technology validation for us,” said Stephane Bancel, Moderna’s CEO, one of the speakers tonight at the Xconomy event titled “Boston’s Life Science Disruptors.”

If Moderna is successful in getting its mRNA drugs through clinical trials, it could radically change the way public health officials think about pandemics. Today, most officials think of combatting these threats with vaccines, which stimulate antibody production through different means. Vaccines can be highly effective, but they often suffer from limited manufacturing capacity and long production cycles. That means it’s usually not practical to react nimbly, and scale up massive production, during a fast-moving pandemic. Similarly, there are a few injectable antibody drugs that officials can turn to in that kind of situation, but those products, including one from Baxter Healthcare derived from human blood plasma, also face manufacturing cost and capacity constraints.

The idea of using mRNA drugs is quite different. These are synthetic compounds that can be designed and manufactured in just about three weeks, with a customized genetic sequence for making any protein of interest. Theoretically, a new pathogen could be discovered, scientists could sequence the new genome in a day, and turn around almost immediately with a sequence for an mRNA molecule that makes antibodies to fight the bug. Scaling up the manufacturing is relatively easy, because mRNA drugs are made through a common chemical process. These compounds don’t need to go through the time-consuming process of being incubated in living cells and purified, like how today’s engineered antibodies are made.

Once the mRNA drug is injected into a person, it should take no more than 24 hours for the person to start producing the protective antibody that’s desired. So, at least in theory, a completely new and deadly strain of flu could be identified one day, and millions of people could … Next Page »

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