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With New Startup, Caperna, Moderna Gets in on Cancer Vaccine Buzz

Xconomy Boston — 

Moderna Therapeutics remains the Boston area’s most mysterious, yet well-capitalized private biotech, with more than $800 million to play with. And while it’s still unclear just what type of progress the messenger RNA specialist has made scientifically, it’s just put more of that cash to use today by christening its fourth drugmaking subsidiary.

The new startup, Caperna, will focus solely on mRNA vaccines for cancer. Moderna calls these “personalized” cancer vaccines that would be used in tandem with other immunotherapy drugs, like the “checkpoint inhibitors” that spur on the immune system to fight cancer.

Like Moderna’s three other startups—Valera, Onkaido, and Elpidera—Caperna is an entity incubated within Moderna that will operate as a wholly owned subsidiary, with access to the Moderna bankroll. CEO Stephane Bancel (pictured above) has said before that Moderna has put about $20 million in cash into each of these companies to start, and added today that it’s already “doubled down” on Valera because of the progress it’s made. That’s easy to do for Moderna—it currently has $840 million in the bank, according to Bancel.

“It will be the exact same with Caperna,” he says.  “We’ll just keep plugging cash [in] as we think is appropriate.”

Moderna has launched this company at a time when cancer vaccines have started to gain traction once again. Over the past month, two companies—Neon Therapeutics and Gritstone Oncology—were launched with significant Series A rounds, both aiming to develop personalized cancer vaccines of their own. Neon and Gritstone aim to do this by identifying what are known as “neoantigens,” small protein fragments that are unique to tumor cells and can tip the immune system off that a cancer is growing. The presence or lack of neoantigens is believed to be one reason folks respond, or don’t, to checkpoint drugs. The idea behind these companies is to analyze peoples’ tumors, find the key neoantigens, engineer synthetic versions of them, and load them into a vaccine—which hopefully trains the immune system to spot the cancer.

Moderna aims to do something similar, just faster, and according to Bancel, in a way that could lead to a more significant immune response. For those unfamiliar, the company is trying to develop a completely new class of drugs, synthetic versions of mRNA—the molecules that carry genetic code to the part of the cells that manufacture proteins. These mRNAs are meant to enable patients to make their own therapeutic proteins inside their bodies.

Bancel says that through the work its infectious disease startup Valera has done, Moderna has seen “extremely strong T cell responses, unlike anything we’ve seen before using proteins or peptides [with vaccines].” He also says that these responses have occurred far faster than with typical peptide vaccines. “Peptide vaccines usually take four, five or six weeks to get enough antibodies to start [making an impact],” he says, adding that Moderna—in preclinical studies—has seen that happen “after only one week, and only one dose.” So Moderna has split off that cancer vaccine work and put it into Caperna.

Bancel says the company is trying to figure out exactly what an mRNA cancer vaccine would look like. Can Caperna put 15 mRNAs into the same vaccine, for instance? “All that work is ongoing and is still being optimized,” he says.

Like everything else with Moderna, this all remains to be seen. Despite the company’s meteoric rise—it has been able to raise all that cash from venture financings and partnerships in just a few years—little is known about the scientific progress it has made. That’s led to some skepticism about what Moderna will ultimately deliver, and that skepticism only increased last week when FierceBiotech reported that Joseph Bolen, Moderna’s chief scientific officer and head of R&D, had left the company.

Bancel says that Bolen left because “he felt that the job was not the job that he came in two years ago to do.” Moderna has evolved into part biotech, part startup incubator, and in the process has gotten more structurally complex. Moderna now has a chief medical officer, Tal Zaks, Stephen Hoge is running its mRNA platform, and each venture is doing “most of the biology heavy lifting for each therapeutic area,” according to Bancel.

“I would have preferred for Joe to stay, I tried to convince him to,” he says. “But he wanted to do something different which is of course his decision.”

Some answers about Moderna’s progress, meanwhile, could finally come next year. Bancel has said before that the company should have a number of mRNA drugs in clinical trials by the end of 2016, and will begin sharing data with the scientific community then as well—Moderna has purposely kept things close to the vest to protect its intellectual property and trade secrets. That’s why the company still has no intention to go public, despite all of its “crossover” investors and financial might.

Bancel reiterated today that Moderna’s development plans remain on track, and that the company currently has several programs in the preclinical studies needed to prepare for clinical trials.

Valera, for instance, is working on 12 programs—five in animal testing via an alliance with Merck—and looks like it’ll have the first of Moderna’s mRNA drugs to reach a clinical trial, according to Bancel. That’s even though Onkaido, an oncology-focused startup, was created in January 2014, a full year before Valera.