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

12/6/12Follow @gthuang

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change the world. “He’s every bit an entrepreneurial startup guy in character, mindset, and approach,” Afeyan says.

Indeed, Bancel adds, “We’re going to rewrite the book of biotech on this company. Everything’s going to be different. We’ll do nothing by the playbook.”

Make Your Own Drug

With any such bluster, skeptics are sure to abound—as well as some confusion. The mere presence of any kind of “RNA” in the company’s approach might make some observers think of RNAi and other techniques for controlling gene activity. These methods have proven difficult to commercialize and sustain in some cases, and a lot of basic science is still being worked out.

In any case, most of the biotech community will be hearing the truth about what Moderna is doing for the first time today. Since its founding, the company has been in “total stealth mode,” Bancel says. “Because if anybody in pharma got hold of that idea, they’d put 50 people and $50 million on it, and they would kill us.” (To keep things on the down-low, the company also hasn’t corrected the misperception that it was working on stem cells, until now.)

The startup says it is protected by its patent filings—80 of them, according to Bancel, covering everything from the chemistry to manufacturing techniques to specific gene sequences for individual protein drugs. “We went crazy on the IP so that when we come out of stealth mode, we’ve locked our position and we secure the company in a very broad and aggressive way,” he says.

Indeed, Moderna spent a good chunk of its early funding on securing intellectual property, Bancel says. Now it is looking to expand its operations and get down to the business of developing drugs and running clinical trials. The company is recruiting staff and says it currently has more than 25 employees. It will also try to form a few high-quality partnerships with pharma and large biotech companies when the time is right, Bancel says.

Moderna says it plans to develop drugs for rare diseases by itself, while working with big companies that have the resources to develop, test, and sell drugs for larger disease markets such as cancer and cardiology. “A company could partner with us and get five drug candidates,” Bancel says. “They can be in five Phase 1 [clinical trials] in a year from now.”

As the company’s chairman and lead investor, Afeyan isn’t counting his chickens just yet. He points out that the biotech industry has seen many cases of “a lot of excitement followed by disappointment.” He emphasizes that Moderna must show how broadly applicable its technology is, and that it is safe in humans. Still, he says, “It’s quite intriguing.”

Bancel concurs with Afeyan’s cautionary tone. “With biology, until you’re in man, you’re not in man,” he says. “I’m not saying it will [definitely] work. But what if?” He adds, “Here there’s no biology risk. We just make what the body makes.” And if all goes well in clinical trials—yes, still a big “if”—the team should know exactly what its drugs will do, because they will, by design, produce human proteins with the right shape, configuration, and so forth.

“It’s the perfect personalized medicine,” Bancel says. “You make your own drug.”

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.