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.”