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Moderna, $40M in Tow, Hopes to Reinvent Biotech with “Make Your Own Drug”

Xconomy Boston — 

Noubar Afeyan, Bob Langer, Tim Springer, and a host of other Boston biotech luminaries have been sitting on a big secret. Today we’re going to out them.

Moderna Therapeutics, a stealthy startup in Cambridge, MA, is the subject of their considerable brainpower and experiments. The company is finally emerging from stealth, having raised $40 million to date from Flagship Ventures and private investors.

Here’s the idea: Instead of producing protein drugs using genetically engineered bacteria in dedicated manufacturing plants—the dominant biotech model of the past 30 years—why not enable the human body to produce its own protein drugs, on demand?

Much easier said than done, of course. The key is developing a technique that triggers cells to produce the drug proteins, without also making the body’s immune system go haywire. To that end, Moderna (pronounced “mode-UR-nuh”) starts by making a specially modified version of messenger RNA. Inject that messenger RNA into the body—so far the company has done tests in mice, rats, and monkeys—and the cells produce the drug proteins right in the body. (More on this below, but the gist is that mRNA, an important intermediary in the process that cells use to make proteins, can be produced using relatively cheap chemistry, whereas proteins themselves cannot.)

If the process works safely in human patients—always a huge “if” in biotech—it could be the biggest story since, well, probably the rise of Genentech and the entire industry in the late ‘70s and early ‘80s. But there’s a lot to be done before the company gets to that point.

“If we’re safe in man, this is crazy,” says Stéphane Bancel, the CEO of Moderna. “It will change the industry in several ways. One is how fast we’ll be able to bring new protein-based drugs to market, with a manufacturing process that’s much cheaper. Our technology can make any human protein.”

The back-story here is pretty interesting. Moderna’s technology was born in the lab of researcher Derrick Rossi at the Harvard Stem Cell Institute and Harvard Medical School. In 2010, Rossi’s team developed a method for modifying messenger RNA and injecting it into human cells as a way to produce cells that mimic embryonic stem cells, in that they can become any other kind of cell. The key advance: the method did not run the risk of triggering an immune-system response if the cells were used to treat patients. (The original technique for producing such stem cells used viruses, posing a risk of an immune response. It also earned its inventor, Shinya Yamanaka, a share of the Nobel Prize in physiology/medicine this year. Yamanaka’s Nobel lecture is this Friday.)

To commercialize his method, Rossi met with Tim Springer, an immune-disease expert also from Harvard Medical School; Springer is known for founding LeukoSite, which was bought by Millennium in 1999. They then hooked up with Bob Langer, the prolific MIT scientist and entrepreneur, and Ken Chien, a cardiology expert from Massachusetts General Hospital and Harvard Medical School. Together they had an idea to start a stem-cell company. Their first stop: venture capitalist Noubar Afeyan, the managing partner and CEO of Flagship Ventures.

Afeyan (pictured at left) knows his biochemical engineering and proteins, among other things, and he saw something very different in what they were showing him. For one thing, he didn’t want to invest in a stem-cell company—there would be too much biology risk. Instead, Afeyan asked the researchers if they could inject some of their messenger RNA, coded for a human protein (say, EPO, a hormone that regulates production of red blood cells), into a mouse and see if they find the human protein in the mouse’s blood.

Rossi went back to his lab and did the experiment—and it worked. Afeyan and the team recognized the significance of the result and filed … Next Page »

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  • Beth

    This is outrageous!

    • Beth

      I am looking forward to getting my hands on the data. Great stuff!

      • Bill

        Several labs claimed that Rossi publication could not be reproduced.

      • Beth

        Lucky you! The data already exists by researchers at the University of Pennsylvania who discovered this about 6 years ago! K. Kariko

    • Dan

      Don’t believe the hype.

  • it does sound very intriguing and has a bit of thinking out of the box.

  • Jaclyn

    Isn’t this the same technology another company started with in 2009? I thought researchers at the University of Pennsylvania had this idea.

    Here’s the link to a government small business grant.

    • Daavd

      That is shameful. Looks like they have been “make your own drug” for years!

  • Dan

    It’s rather easy to deliver things to the blood, say EPO, but what about delivery to other organ systems? If we can’t get a stabilized siRNA to the tissue of interest, how are going to get an mRNA?

    • Daavid
      • Don

        Didn’t Tekmira sue Alnylam for using those LNPs that Tekmira owns? It looks like Alnylam had to pay $65M and $10M milestone payments. http://www.reuters.com/article/2012/11/13/idUS126737+13-Nov-2012+HUG20121113

        I wonder if Moderna will have to pay the University of Pennsylvania that much for infringing on any patents from the University of Pennsylvania.

      • Dan

        Right. I’m aware of that technology. Unfortunately, it doesn’t work so well either.

    • Evan

      Other companies have used lipid nanoparticles to deliver a range of drugs into people—from chemo-therapeutics like tamoxifen to even some nucleic acid molecules. Usually these lipids formulations come with some pretty serious side effects…and in the case of a nucleic acid things like gout and possible kidney problems from excess uric acid. I guess it depends on how often they have to dose it. But since it’s an mRNA and not stable, probably more than once.

      • Dan

        True, but they don’t work especially well. There’s also a secondary issue of targeting the cells of interest.

  • yeahright

    Show some results.

  • Sidv220

    Nice review though. You can get a real picture of Moderna from its present and former employees at http://www.glassdoor.com/Reviews/ModeRNA-Therapeutics-Reviews-E453959.htm. I would assume its management is over claiming and making claims which could be questionable. My assumption is based upon company review/comments from its employee and former employee. Moderna can never be Genentec but definitely qualify for a company which would disappear from the radar after 2-5 years.