I

t has a license to one of the biggest scientific advances in history, and its rivals have made big money on Wall Street with splashy public offerings. But CRISPR Therapeutics, the quietest member of an exclusive club with the rights to turn gene-editing technology into medicine, seems content to keep its work secret, disclosing little about its targets or timelines. At least for now.

As its name would suggest, CRISPR Therapeutics is working in the booming field of, well, therapeutics with CRISPR —  a powerful genetic tool that allows scientists to find and replace the errant strands of code behind certain ailments. The science behind the technique first came to light just four years ago, but its rapid progression has researchers buzzing about future cures for muscular dystrophy, malaria, and a host of rare diseases.

All three companies that have licensed the technology — Editas Medicine, Intellia Therapeutics, and CRISPR Therapeutics — have their R&D operations in Kendall Square, about a mile apart from each other.

advertisement

Editas and Intellia have boomed since their initial public offerings, creating huge windfalls for their investors. Editas’s backers had paid an average of $2.11 per share before the company went public in February. Today, those same shares are worth around $30 apiece, and Editas is valued at more than $1 billion. For Intellia, its share value has increased about sevenfold since it started accepting private investments, and, after executing an IPO last week, the company commands a market value of about $800 million.

Backers of CRISPR Therapeutics presumably wouldn’t mind a 1,400 percent return on their investment. But if the company is preparing for an IPO, it’s keeping that information to itself. STAT reached out to CRISPR Therapeutics’ top management, its board of directors, and many of its investors, and not one consented to an interview.

Despite its recent silence, CRISPR Therapeutics’ latest big hire could provide a clue as to its intentions. The company recently recruited Marc Becker to be its chief financial officer, noting in the second paragraph of its March hiring announcement that Becker led his last employer, rEVO Biologics, through the IPO roadshow process.

The big benefit of going public is simple: money. You can’t do anything without it in biotech, and companies can raise it more easily on Wall Street than they can in private. After some lean years post-downturn, the investment community has warmed up to the idea of buying into risky drug developers, and more than 140 biotech firms have pulled off IPOs since 2013.

But there are drawbacks. Going public “changes the nature of the beast,” said Paul Leiman, who studies the business law of biotech at the Johns Hopkins Carey Business School. Small companies take on huge administrative burdens when they list their shares, Lehman noted, and the sudden influx of new, opinionated investors can sometimes force management to change its plans.

Furthermore, public companies have to tell the world what they’re doing, while private firms get to pick and choose and their disclosures. Like CRISPR Therapeutics, Intellia and Editas were once cagey about their development pipelines, but in documents filed with the government prior to their IPOs, they had to spell out the what, when, and how of their work.

In biotech, where legal spats over intellectual property are commonplace, operating in secret can create a competitive advantage. Moderna Therapeutics, a Cambridge biotech valued at more than $3 billion, has no intention of going public for that very reason, according to its CEO. Boston’s Intarcia Therapeutics, meanwhile, has remained private through the long, costly process of running clinical trials, and it plans to stay that way, all while maintaining a valuation north of $5 billion.

As for CRISPR Therapeutics, the biotech has put itself in a position where it doesn’t need Wall Street’s money to move forward, according to a former company executive.

Shaun Foy, who was the company’s founding CFO, said CRISPR Therapeutics has enough private cash to advance its internal goals and has cemented relationships with larger drug makers to help out with the projects too big for its team.

To date, CRISPR Therapeutics has raised $154 million through the sale of equity. The company banked another $75 million last October from Boston’s Vertex Pharmaceuticals, which licensed the rights to as many as six CRISPR-based therapies and promised to pay as much as $420 million for each down the road. In December, CRISPR Therapeutics also partnered with Bayer to launch a 50-50 joint venture, and the German pharmaceutical giant has pledged to invest at least $300 million into the effort over five years.

All those dollar signs give CRISPR Therapeutics a long runway and free it from the pressure some cash-light biotechs feel to go public, said Foy, who left the company on amicable terms last summer.

CRISPR Therapeutics began in 2012 as a three-person operation called Inception Genomics, with Foy handling finances, former Sanofi executive Rodger Novak as CEO, and award-winning scientist Emmanuelle Charpentier contributing her game-changing discoveries in the science of CRISPR.

Charpentier, alongside Jennifer Doudna of the University of California, Berkeley, first described key aspects of the cut-and-paste methodology in the scientific literature, and both are embroiled in an ongoing intellectual property dispute with the Broad Institute and the Massachusetts Institute of Technology over who invented it first. Doudna has aligned with Intellia — with CRISPR Therapeutics, by extension, in the same camp — while MIT and the Broad are working with Editas.

In the years since its incorporation, CRISPR Therapeutics has sprouted from a tiny upstart to “a machine with 60-plus people, and they’re growing very quickly,” said Foy, who now runs an Irish company called ERS Genomics, which is tasked with licensing Charpentier’s work for research and other nontherapeutic applications.

Whether CRISPR Therapeutics’ radio silence means the next step is an IPO remains to be seen. In the past, the company has downplayed big questions about its future, with Novak, who remains the CEO, telling STAT in January that the process “is not a sprint, it’s a marathon.”

But his investors, seeing the returns from CRISPR IPOs elsewhere, might be asking him to pick up the pace.

Elie Dolgin contributed reporting.

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

Newsletters

Sign up for The Readout: A guide to what's new in biotech

Please enter a valid email address.

Leave a Comment

Please enter your name.
Please enter a comment.

Sign up for our Morning Rounds newsletter

Your daily dose of news in health and medicine.

X