Synlogic has already attracted some $35 million from investors on both coasts, including the Bill & Melinda Gates Foundation, to pursue a science fiction-like idea to make customized disease-killing bacteria. Now, the Cambridge, MA-based startup has brought in a well-known former Pfizer executive to lead its first programs into clinical testing.
Jose-Carlos Gutierrez-Ramos today is being named the president and CEO of Synlogic, a startup formed by Atlas Venture and New Enterprise Associates last July. (The Gates Foundation invested in the company a few months later.)
The announcement comes two weeks after Gutierrez-Ramos (pictured above) stepped down as the senior vice president and head of Pfizer’s Cambridge-based Biotherapeutics R&D to “return to the biotech sector,” as Pfizer noted at the time.
He’s taking over for Synlogic acting president Ankit Mahadevia, a venture partner and entrepreneur-in-residence at Atlas.
Gutierrez-Ramos has spent more than a decade in various executive roles at several pharma companies. He’s coming off a five-plus year run at Pfizer, with stints at GlaxoSmithKline, Amgen, and Millennium Pharmaceuticals before that.
The challenge here is far different than Gutierrez-Ramos faced in his Big Pharma days. At Synlogic, a 15-employee startup, he’ll try to develop what amounts to a new type of drug—manmade microbes that are programmed to sense a specific disease or infection, secrete a drug to treat it, and then self-destruct when they’re done.
The concept, which comes from the work of MIT’s James Collins and Timothy Lu, is an offshoot of what’s known as synthetic biology, the field of research into genetically engineered biological parts and systems. Synthetic biology has had a lot of hype over the years; Collins himself noted at a recent Xconomy event that the field made its share of mistakes, serving as a “cautionary tale for investors.” (Check out this recent profile in Newsweek, for instance, on the synthetic biology industry’s issues producing biofuels on a large scale.)
Gutierrez-Ramos acknowledges that synthetic biology hasn’t delivered in healthcare yet. But he’s been following the field closely and has been itching to be a part of it. He cites key advances—scientists, for instance, have learned what can and can’t be done with synthetic genetic circuits, he says: “They’re much more developed than years ago.”
Synlogic is an example of that. Think of its potential therapies as “smart” drugs; tiny bugs engineered logically, like a computer program designed to respond only to specific disease scenarios, and equipped with various fail-safes to make sure they stop when their job is done. If they were to work—the first likely won’t be tested in humans until at least late 2016 or early 2017, Gutierrez-Ramos says—the presumed benefit would be greater control of safety and effectiveness. A custom-designed drug would spare healthy tissue and only attack if the right conditions are present.
Gutierrez-Ramos says these therapies should be cheaper and quicker to develop than normal drugs; he estimates 12 months from scratch to a development candidate. That’s in part because once the genetic circuitry is in place, it only takes Synlogic “weeks” to have a new bacteria or virus to test against a different disease. He likens the process to fine-tuning an engine that’s already been built, rather than scrapping it and building a new one.
“I just think this is going to change the way we think about therapeutics, I really do,” Gutierrez-Ramos says. “And I want to be part of that, I want to lead it.”
Given this is a new, unproven technology, Synlogic’s strategy is to start out going after a few rare diseases, where the “benefit-to-risk ratio is extreme,” and the company can know quickly whether it’s onto something, he says. Its first two candidates will target urea cycle disorders (UCD), the name for a group of genetic diseases that trip up the body’s waste-disposal system; and phenylketonuria (PKU), an inherited disease characterized by the abnormal buildup of an animo acid called phenylaline, leading to mental impairment and other health problems.
Every year, about 14,000 patients in the U.S. are diagnosed with PKU; and 8,000 with a UCD. The available treatment options, which mainly consist of strict diets and multiple pills per day, aren’t very effective.
The goal is to keep ownership of the smaller, less capital-intensive programs and form partnerships with big companies for diseases that require large, expensive clinical trials. Gutierrez-Ramos says that Synlogic has several term sheets in hand for different disease types, though he wouldn’t specify. Those potential transactions could shape where Synlogic goes from here.
“Depending on how much non-dilutive capital we raise, how the markets are, what happens to our lead programs, we’ll see what the next step is,” he says.