With a crucial CAR-T trial looming for Cellectis, a confident André Choulika is laying it all on the line

Cellectis CEO André Choulika

CHICAGO--André Choulika does not mince words.

The Cellectis CEO is an outspoken champion of all things related to the company he founded 15 years ago. Beirut-born, with a Ph.D. from the University of Paris and research stints in genetics at Harvard and Boston Children's behind him, Choulika doesn't hesitate for a moment when it comes to dismissing the technologies and targets of rival biotechs that have raised hundreds of millions of dollars in the past two years as so many amusing novelties, or at best niche players, that will be largely sidelined once Cellectis makes it past the finish line.

Based on the common wisdom of Wall Street, Cellectis ($CLLS) is in the second wave of companies focused on CAR-T, using its gene-editing skills to create allogeneic, or off-the-shelf, T cell therapies that can fight cancer. First up, most analysts would say, will be Novartis ($NVS), allied with researchers at Penn, Kite ($KITE)--collaborating with a top researcher at the National Cancer Institute--and Juno ($JUNO), which is racing ahead with research from Memorial Sloan-Kettering and the Fred Hutch. They are adapting patients' T cells to create personalized, autologous therapies, blueprinting registration studies with an eye to grabbing first-mover rights from the FDA.

But that's not how Choulika sees it.

"Cellectis is the first company doing CAR-T," the CEO told FierceBiotech during an interview at ASCO. "We are the first gene editing company in the world," dating back to 1999. "There was no gene editing before us; we are the leaders."

Those aren't just idle boasts. Choulika was a pioneer in his use of meganucleases in gene editing. He's now devoted his company to a gene editing technology called TALENs to engineer cells--looking to create the maximum efficiency in terms of cell expansion inside patients.

But there are several editing tools to choose from. Juno recently tied up with Editas, one of a handful of upstarts now championing CRISPR/Cas9 gene editing tools, the same approach Johnson & Johnson's ($JNJ) Janssen plans to use. And Sangamo uses zinc finger nucleases to do its gene editing work.

Choulika, who needs to prove that Cellectis has the gene-editing skills necessary to fashion a ready-to-use CAR-T product that won't spur a potentially lethal immune response, is going his own way with TAL tech in-licensed several years ago. And the new wonder technology CRISPR/Cas9, he says, can't compete.

"Any stupid guy can design a CRISPR, even Brad," he says, pointing to his PR man sitting nearby and flashing another grin. "It's cheap to design and quick to design; one week and 10 bucks. Any academic researcher can order CRISPR online and become a gene editor."

But it's not nearly as precise as TALENs. Says Choulika: "Its efficiency is really bad."

Choulika is even more dismissive of his autologous rivals. He scoffs at the idea that a personalized T cell treatment can make a good product.

"It's a service, not a product," he says. And that doesn't make for much of a business model, says the CEO, who likes to talk about 85% and 90% gross margins on what he plans to sell.

The autologous strategy "is a therapeutic approach made by academics," with "flabbergasting" outcomes "but not an industrial approach. We're a commercial company," he adds, with plans to make money, a lot of money, while benefiting patients and rewarding investors.

Eventually, he says, the only reason why autologous cell products will be made will be to handle the marginal number of patients who don't respond to an off-the-shelf product like his.

There is one major consideration about Cellectis' therapeutic approach that Choulika takes on with equal frankness: "We don't know if it works," he says simply.

All of Cellectis' work in this field up to now involves preclinical research. A small, absolutely critical, human study will be launched soon to put this technology to a human test. And the future of the company is riding on the outcome.

Does that cause any sleepless nights? Choulika shrugs off a nagging question like that with a big smile.

What happens if it doesn't work? he says. "I won't have cancer."

He'll survive, just as he has before.

"I try to disconnect all this," he says. "I try to do good things. I've experienced very bad decisions that I made and paid dearly for them. I'm always surprised. I'm like a kid, always surprised and always predicting things. On the short term, I'm not a good predictor, but I'm not bad predicting on the long-term. If I pulled a business plan from 15 years ago, it's still active today. We're in the hottest field, gene editing. We're leaders in gene editing. By far there's not a single company that compares. I've been 28 years in gene editing. But in the short term I'm always startled. CRISPR startled me; Juno with its $150 million Series A, startled me. Companies like bluebird bio don't surprise me. It's a fantastic company--impressive but not surprising. I predicted last year that everything will turn to gene editing."

Positioned at a sizzling hot tech crossroads involving CAR-T and gene editing, Choulika has made plenty of believers around the world. That kind of potential attracted investors to a Nasdaq IPO that raised $228 million, giving the company a current market cap of $1.4 billion. It was no great surprise to see the Financial Times come back with a story late last week that Pfizer ($PFE), which partnered with Cellectis, is pursuing talks to acquire the company.

But he isn't telling FierceBiotech anything about that. "I don't respond to market rumors," says the CEO with the kind of instant comeback that comes with practice. He's much more voluble, though, when it comes to highlighting plans to build a CAR-T R&D center at the growing biotech hub on Manhattan's East Side where he now has a staff of 12.

It is, by his account, the absolute best place in the world to do what he wants to do.

"They don't like me to say this, but New York is like the business center of the world, flights from all over the world, and not just one a day. It's easy for me to come to New York. The second thing, New York has a powerful position to build a biotech hub, so they're helping companies. The cost is way lower even in Manhattan."

"Our goal is to build it up." And Cellectis is hiring M.D.s and Ph.D.s with an eye to creating a major CAR-T player with multiple products.

"The goal is to expand, but I have no limit," says the CEO. "My goal is to find the best people ever."