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

12/6/12Follow @gthuang

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their first patent shortly thereafter. And so Moderna was born, with Rossi, Afeyan, Chien, and Langer as its co-founders. (Springer is on the board of directors.)

Afeyan is cautiously optimistic about the startup’s chances. “It definitely could enable a resurgence of a new type of biotech industry and company,” he told me earlier this year. “It certainly has a disruptive potential. And it builds on lots of things that have been tried before.”

Rewrite the Book of Biotech

Since the Genentech days, the biotech industry has made protein drugs by taking cells that grow well in culture—typically bacteria, yeast, or mammalian cell lines—and inserting a human gene that codes for a particular therapeutic protein. Grown in big vats, the cells produce the desired protein (insulin, say), and a company can purify it into a form that can be put in a syringe and injected into patients. That’s the idea behind protein drugs from Amgen, Baxter, Genzyme (now part of Sanofi), and all the other big-name biotechs.

Yet, after all this time, the entire biotech industry has yielded only about 100 approved drugs on the market. Everyone in the industry knows the immense amount of time and resources it takes to formulate and fine-tune the production of a new protein drug. By contrast, Moderna has already looked at dozens of proteins—some secreted in the blood and some intracellular (expressed inside cells). As Bancel puts it, “We can make any therapeutic protein in a matter of weeks.”

That’s because, if it really works, Moderna’s technology will let the body’s cells produce the proteins themselves—in response to the right messenger RNA. The company’s process for making mRNA is fast and compact, Bancel says. It buys nucleotides altered by a special chemical process—that’s the trick—and uses a catalytic process to create many copies of the messenger RNA, so that a tank of material will fit in a coffee cup, he says.

At a high level, this sounds a bit like what researchers were trying to do in the early ‘90s, in experiments that eventually led to the discovery of RNA interference. They were trying to make flowers more purple (and other colors) by adding an mRNA for a “purple” gene, but they wound up making them whiter instead—meaning the gene expression was inhibited instead of boosted. Bancel emphasizes that his company’s technology, if it’s related at all, does the opposite of RNAi. But exactly what makes Moderna’s mRNA work so differently is under wraps.

Bancel (left) joined the company as employee number two last year, after stepping down as CEO of bioMérieux, the French diagnostics company. He says it took a lot to get him to leave his previous job. “I wanted to start a company from scratch,” he says. “I turned down many, many projects because the potential was not big enough.”

But Bancel’s background was a good fit for Moderna because he was a biochemical engineer by training, had experience manufacturing drugs at Eli Lilly, and had run a large, publicly traded company in bioMérieux. And he was still hungry to … Next Page »

Gregory T. Huang is Xconomy's Deputy Editor, National IT Editor, and the Editor of Xconomy Boston. You can e-mail him at gthuang@xconomy.com or call him at 617-252-7323. Follow @gthuang

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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.

  • http://www.engag.io/Abdallah Abdallah Al-Hakim

    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.
    http://www.sbir.gov/sbirsearch/detail/294079

    • 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.