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find themselves able to fend it off with protective antibodies stirred by mRNA within a month.
Other biotech companies with RNA-based drug technologies have secured big support from the U.S. military. Cambridge-based Sarepta Therapeutics (NASDAQ: SRPT) is working on experimental drugs for the Marburg and Ebola viruses, as does Vancouver, BC-based Tekmira Pharmaceuticals (NASDAQ: TKMR). But those technologies work in different ways than Moderna’s.
Mike Houston, the former vice president of chemistry and formulation at Bothell, WA-based Marina Biotech, said he was “amazed” that Moderna has shown it can use mRNA molecules to produce not just small, relatively simple therapeutic proteins, but also larger, more structurally complex molecules like Y-shaped antibodies. Houston, now a consultant in the RNA therapeutics field, is familiar with Moderna’s work because he interviewed for a job there earlier this year, but chose not to move his family across the country to join the company.
Making mRNA drugs to coax antibody production “is really important because the time it takes to make an antibody from scratch is really long,” Houston says.
“We’ve had to go as an industry from chimeric human monoclonal antibodies to fully human monoclonal antibodies. Now you’re essentially bypassing all that by having the mRNA and injecting it into the human so they make the antibody themselves,” he says. “It’s really cool. It’s like we’re in the future almost. This blows my mind.”
Besides its scientific and public health implications, the government work could provide a meaningful secondary revenue stream for Moderna’s business. DARPA is providing the grant to develop the technology, and to help Moderna work on mRNA drugs against three specific infectious diseases that Bancel said he can’t publicly identify. DARPA has further agreed to pick up the tab for clinical trial expenses should any of the Moderna molecules get that far. If Moderna can pass all the necessary trials in human beings, it will still own the intellectual property, and could be in position to sell stockpiles of its mRNA drugs to the government, which may want to keep them in freezers just in case they’re needed to fight a pandemic. Or, it could hire Moderna to turn on a dime and start making mRNA drugs against a newly identified pathogen.
Bancel wouldn’t say what the biggest technical challenge is that Moderna must solve to fulfill the DARPA grant. But in the past, he has noted that Moderna must make sure its compounds don’t provoke any nasty immune system reactions—what’s known as immunogenicity. On a recent visit to his office, he also discussed how dosing must be figured out for various mRNA drugs. That’s because tiny doses of mRNA can prompt the body to produce large amounts of therapeutic proteins—perhaps more than necessary or desirable in some situations.
With the validation of AstraZeneca and DARPA, Bancel has options. He almost surely could whip up an IPO road show and take Moderna public if he wanted to, given the investor appetite for new offerings this year. But he has demurred, saying the main goal is to get strong assurance that the compounds are safe before they go into clinical trials. A stumble by Moderna there could be devastating to this entire new area of biology, he says.
“The last thing we want to do is rush and make a mistake,” Bancel says. “We have a big responsibility to society. We believe we can bring forward new drugs to patients. Remember gene therapy? They rushed it to the clinic in the ‘90s, and an 18-year-old died in a clinical trial. It set back the field for a decade. mRNA is brand new, and we don’t want to damage it—it’s our responsibility to society. We are being very responsible and careful, especially now that we have cash. It would be criminal to make a mistake now. We still don’t know what we don’t know. It’s science, so we can never check every box, but in terms of improving product purity, integrity, so that we go in [to clinical trials] knowing as much as we can, it’s very important to us.”