A Big Bet That Gene Editing Will Cure Human Disease

Huddled with Wall Street investment bankers outside a hotel ballroom in downtown New York last January, Katrine Bosley was getting some sobering news. The stock market, falling since early December, was continuing to dive, with her industry, biotechnology, especially hard hit. The previous year had been the worst for new stock offerings in the history of the tech-heavy Nasdaq, and now Bosley had to persuade the roomful of investors lunching on the other side of the wall that her company, Editas Medicine, could break the trend.

Editas specializes in a process called gene editing using a technology known as CRISPR/Cas9. Because this technology makes it possible to edit DNA precisely and more easily than earlier methods, it has quickly become popular with scientists in the four years since it was first described. Editas was cofounded by several of the early developers of CRISPR. Now it aims to find ways to transform this tool into a method of treating genetic diseases that for the most part have no treatments today. Editas is researching therapies aimed at eye disease, cancer, sickle-cell anemia, and Duchenne muscular dystrophy, among others.

Outside scientific circles, CRISPR is still far from widely understood, and Bosley had spent much of her time since joining Editas in June 2014 pitching venture capitalists and possible investors on its potential. Even so, the investors in that New York hotel had plenty of questions about the company—and some remain even after Editas’s highly successful initial public offering, which raised $109 million in February.

The company has no revenue from product sales and expects none in the foreseeable future. It has more than $200 million in cash, enough to last two years, but must in that time create a product unlike any that has ever existed before. And it won’t begin to test its treatments on humans until sometime next year. Even if that is a success, it is unclear when it will be commercialized.

What Editas has going for it—besides Bosley and a premier group of scientific founders—is the potential for gene editing to fundamentally change medicine.

“It’s such a powerful tool,” says Jim Flynn, managing director of Deerfield Management, which runs $5 billion in health-care investments, including a stake in Editas. “There are people here who believe that when we look back in 10 years we will see a demarcation: before gene editing and after.”

Editas cofounders George Church and Feng Zhang were the first to show that CRISPR could work with human cells. Hoping to exploit the technology’s commercial possibilities, a trio of health-focused venture firms in the Boston area convinced Church, Zhang, and others to form Editas only months after their papers were published in scientific journals. Editas isn’t the only company looking to develop gene-editing treatments. Rival Intellia Therapeutics, with which Editas is involved in a patent dispute, went public just three months after Editas. Last December, Bayer agreed to invest $335 million in a joint venture with yet another company, CRISPR Therapeutics.

Bosley has said Editas will be far enough along to begin testing in humans next year. The plan is to inject a virus containing Cas9 into the eyes of people suffering from a rare form of progressive blindness caused by a specific gene mutation. The enzyme would then cut the faulty sequence, triggering a natural DNA response in which the cell repairs the deficit itself. Starting with this eye disease has some advantages, because its genetic basis is clearly understood and the treatment can be delivered locally to a target.

Still, significant technological challenges remain. Editas must make an effective treatment, ensure it can reach the site of the disease safely, and edit the DNA properly—all without dangerous side effects.

The company also has to hope that ethical questions about some potential uses of CRISPR do not cloud the technology and delay regulatory approval. For example, some scientists are concerned that the technology could be used to engineer human embryos. Though this is not something Editas is working on, ­Bosley has met with groups from French members of Parliament to U.K. bioethicists to discuss that and other topics.

She says the best advocates for the technology are patients with the untreatable genetic diseases Editas hopes to address. For them, effective gene editing could be a crucial medical breakthrough. Now Bosley has to prove that Editas can develop something that fits that description.