Engineered mRNA Company Moderna Launches 4th Spinoff, Caperna

Engineered mRNA Company Moderna Launches 4th Spinoff, Caperna
October 22, 2015
By Mark Terry, Breaking News Staff

Cambridge, Mass.-based Moderna Therapeutics, announced the launch of its fourth venture today, Caperna LLC.

Moderna’s business model is built on developing new drugs using its messenger RNA (mRNA) drug platform. Engineered mRNA is potentially capable of making the body’s own cells produce whatever protein the mRNA is programmed to code. Essentially, the concept is to create mRNA molecules that can be injected into the body, where they will then use the body’s molecular machinery to produce whatever drug or molecule it’s programmed to treat. To date, Moderna has then spun off subsidiaries to focus on specific disease states utilizing the mRNA platform.

So far, the company has launched Onkaido, which is focused on oncology, Valera, focused on vaccines and antivirals and anti-bacterial agents, Elpidera, for rare diseases, and now Caperna. Caperna will focus on cancer vaccines.

“With such a potent vaccine platform and a manufacturing process that lends itself directly to rapid production of patient-specific therapies, we believe Moderna’s mRNA approach will offer distinct advantages in the development of new cancer therapies,” said Tal Zaks, chief medical officer of Moderna, who will act as interim president of Caperna, in a statement. “We expect our ability to specifically activate and direct the immune system will synergize with checkpoint inhibitor therapies like PD-1 antibodies.”

Moderna was founded in 2011. In January 2015, the company indicated it had raised $450 million in a private investment round, which is believed to be the largest for a privately held biotechnology firm. Since its founding, the company raised $950 million in venture capital and non-dilutive funding. Apparently the company isn’t particularly interested in going public. Investors have included Viking Global Investors, Invus, RA Capital Management, and Wellington Management. Its founding investor is Flagship Ventures.

“We were clear with our investors from the get-go that we’re playing the long game,” said company chief financial officer Lorence Kim to Xconomy in January. “A typical crossover is done with an expectation that an IPO is just around the corner. And we were very clear with people that that’s not the case here.”

The company indicated that at that time it had 45 separate preclinical programs, 29 of them through a partnership with AstraZeneca . It also had developed seven programs through an agreement with Alexion. In 2013, it received a grant from the Defense Advanced Research Projects Agency (DARPA) worth $24.6 million over five years to work on infectious diseases.

As intriguing as Moderna’s technology platform is, it’s still largely unproven and hasn’t moved into human clinical trials yet. In January, Stephane Bancel, Moderna’s chief executive officer, said, “The early signs in animals are quite exciting. We might be able to do a few more things with mRNA than we ever even thought in our wildest dreams.”

Typically the problem with these types of technologies is evading the body’s own immune system. If Moderna has licked that problem and the technology can be used safely and effectively in humans, it has the potential to revolutionize drug development and drug manufacturing.

Certainly many in the industry are betting on it. In addition to AstraZeneca, Alexion and DARPA, Moderna has developed partnerships with Merck & Co. , Institut Pasteur, Karolinska Institutet, and Karolinska University.

In May 2015, the company was ranked #1 on CNBC’s Disruptor 50 List. “Our mRNA technology is fundamentally disruptive, enabling us to do things not possible — or not done well enough — with current approaches,” Bancel said in response to the award.

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