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Biotech is a risky business, where roughly 90 percent of grand ideas fall flat and billion-dollar failures are commonplace. So it’s fitting that the industry’s self-appointed spokesman is the blackjack-playing CEO of a fast-growing company, one about to find out whether its futuristic science can make an actual product.

John Maraganore is unafraid of the spotlight. The 53-year-old head of Kendall Square’s Alnylam Pharmaceuticals is a regular on CNBC, appearing on camera in his characteristic designer glasses and colorful suits. Mostly he talks about his company’s efforts to develop gene-silencing drugs, but he doesn’t hesitate to defend the virtues — and condemn the sins — of his whole industry, even though his company has yet to win marketing approval for a single product.

Last year, when Martin Shkreli’s Turing Pharmaceuticals raised the price of an old drug by more than 5,000 percent, Maraganore was one of the first biotech executives to castigate the move.


Biotech is “about innovation, patients, and 21st-century medicines,” he said on live television, not “repricing drugs from the 1950s to make a profit.”

It wasn’t a calculated statement, Maraganore told STAT. He got the invitation as he was preparing his kids for school, and he was on the air an hour later, speaking on behalf of drug companies around the country.


“If there are actors in the biotech community out there poisoning the well by creating a negative environment, it hurts us,” Maraganore said in his Cambridge office, sitting beneath a Fiat-sized poster of a drug he developed at another company. “It hurts Alnylam. It hurts any other development-stage company.”

Maraganore’s strongest words, however, are often saved for Alnylam’s competitors.

Last year, when rival firm Arrowhead Pharmaceuticals acquired some gene-silencing assets from Novartis, one of Alnylam’s former partners, Maraganore dismissed the deal as “buying a bag of stale potato chips.”

And that’s what he’ll say on the record. Within Alnylam, he tends to be more candid.

“He will say exactly what he thinks about something someone else did, and people love that,” said Rachel Meyers, the company’s vice president of research. “But there’s been many, many times when the eyes go around the room, and everyone looks at each other and thinks, ‘OK, did he just say that?’”

It’s a risk, as CEO of a publicly traded biotech, to weigh in on issues outside your company. In the drug industry, stocks often rise and fall on rumor and conjecture. Speaking unguarded in a fickle market can seem like tempting fate.

But Maraganore said he’s never heard complaints from other company executives, and Alnylam’s investors or board members have never asked him to tone it down.

He’s been “a very positive force in the whole biotech community,” said Phillip Sharp, a molecular biologist at the Massachusetts Institute of Technology who cofounded Alnylam in 2002 and continues to sit on the company’s board.

And people around the industry, including executives at rival firms, all praised his ability to put a human face on biotech.

Dr. Sean Harper, head of research at biotech giant Amgen, said Maraganore has helped raise the industry’s profile. And Dr. Jay Bradner, the top scientist at Novartis, called Maraganore a “legend” whose decade-plus dedication to Alnylam demonstrates “the most credible type of scientific leadership.”

Today, Alnylam is worth about $6 billion, employs more than 400 people, and pays its CEO about $8.3 million in cash and stock. But when Maraganore left the cancer drug maker Millennium Pharmaceuticals to become Alnylam’s CEO in its early days, the company had just a handful of employees and a few million dollars to its name.

Kevin Starr, then Millennium’s chief financial officer, thought his friend was taking too big a risk: Alnylam’s bedrock technology had been around for just a few years, a blip in the world of science, and the company had only data from mice to back up its ambitions.

But Starr, now a member of Alnylam’s board, soon realized the “nerdy scientist” he once knew had grown into a business leader, he said.

And the change was visible. Maraganore, as former Millennium CEO Mark Levin put it, had gone from a lab dweller with “khaki pants halfway up his chest” to something “more like a model out of Versace than someone out of Boys’ Life.”

Maraganore’s risk-taking streak dates back to his pre-Alnylam days at Millennium, where he was an executive vice president quite fond of making friendly bets. Levin remembers a particularly lucrative meeting in 2000, when Maraganore, unprompted, made the bold assertion that, every 100 years, the month of February stretched to 30 days.

“And everybody’s like, ‘John, that’s not right,’” Levin said.

But, this being a presmartphone world in which such claims could not be instantly checked, Maraganore held onto his version of planetary science, and he dared his colleagues to put some money on it.

Maraganore lost $1,000 that night, recalled Levin, who together with Starr and another Millennium alum went on to launch and run Third Rock Ventures, a Boston firm dedicated to bankrolling startups.

Maraganore’s most recent gamble, betting on an unconventional approach to treating disease, is looking more promising.

Biotech companies have long developed humanized antibodies that block the errant proteins behind many diseases. But Alnylam’s technology goes one step upstream, tinkering with RNA to stop those proteins from being produced in the first place.

Now, after spending about $1.4 billion over 14 years, Alnylam has reached the final stage of development with its top prospect, a treatment for a rare, life-threatening disease that results from the buildup of misfolded proteins. And the company is so confident the drug is going to work that it just broke ground on a huge manufacturing facility south of Boston, which is slated to come online in 2018 — right when Alnylam expects to win regulatory approval.

“We’re making those all-in decisions right now,” Maraganore said.

Both Levin and Starr said they continue to see Maraganore’s growing appetite for risk first hand each and every year: All three make an annual pilgrimage to Las Vegas for a weekend of marathon blackjack games with a few hours of sleep peppered in, a tradition that dates back 13 years.

They all bet “big stakes,” Maraganore said, though he declined to disclose just how those bets pan out.

“You know, I’m pretty break-even,” he said.

Kendall Squared brings you dispatches from the world’s epicenter for biotechnology and drug discovery.