Out on the cutting edge of gene therapy, Editas Medicine nabbed a huge round of early funding. Now it has nabbed one of the highest profile CEOs of the Boston biotech scene.
Katrine Bosley and Editas said today she is the Cambridge, MA-based firm’s new chief executive, her first full-time role since she led Avila Therapeutics into a big buyout from Celgene two and a half years ago. She’ll take over for Editas’ interim CEO, Kevin Bitterman of Polaris Partners.
It’s a formidable project. Editas is one of only two companies that have publicly set their sights on turning a brand-new genome editing technology, known in shorthand as CRISPR-Cas9, into therapies for patients with genetically driven diseases. Like RNA interference before it, CRISPR is a stunning breakthrough in understanding basic biology, and it has caught on as a widely used biomedical research tool to quickly edit the genes of animals used to study disease.
“This science captures the imagination,” said Bosley. “It has moved so quickly because it works [as a research tool]. Now the next challenge is to turn it into therapeutics. There are mountains to climb to make that happen, but we’ve got a compelling foundation.”
The field of gene therapy, which lay nearly dormant after a volunteer in a clinical trial died in 1999, has in the past few years been rejuvenated—or “derisked,” as people in the drug industry like to say. One product has finally been approved in Europe, and several programs from various companies are holding promise in human trials, including one from Bluebird Bio we reported recently.
However, Editas, and another recent startup, CRISPR Therapeutics in London, are not nearly as far along, and making bets on much less validated technology. The idea behind CRISPR-based treatments is to essentially perform gene surgery: sending an enzyme, nature’s molecular scissors, into a cell’s nucleus with an RNA guide to seek out and snip out a defective gene, and perhaps even replace it with a new, functional strand of code.
Whether Editas can make the technology work, of course, remains to be seen. One big problem will be delivery: How to get the enzyme and its RNA guide into the right cells. Another problem is specificity, or being certain that the CRISPR-Cas9 complex isn’t editing genes in unwanted places. “It’s one thing to have the CRISPR apparatus go where you want it to go,” said Bosley. “But we need to know does it go anywhere else?”
Another shadow looming, as with so many new technologies, is intellectual property. Sangamo Biosciences (NASDAQ: SGMO) has made clinical progress with its own gene-editing technology known as zinc finger proteins, and it has made more expansive ownership claims about the gene-editing field in the past.
And Emmanuelle Charpentier, the French researcher whose work was critical in turning CRISPR, a once-obscure niche in bacterial science, into a therapeutic possibility, has thrown her weight—and her intellectual property—behind CRISPR Therapeutics. That firm is backed by Versant Ventures.
Another company, called Caribou Biosciences, has licenses to some of Charpentier’s work, though it’s said publicly that it’s currently focused on gene editing tools, rather than therapies.
Versant’s CRISPR Therapeutics is building a platform around its technology and plans to spin out therapeutic programs into separate entities. It’s a model that has caught on among biotechs in recent years as a way to create simpler structures for investors and cleaner divisions between therapeutic areas. Another Cambridge startup with a cutting-edge platform, Moderna Therapeutics, recently told Xconomy that it has quietly switched to a similar spin-out strategy. When asked if Editas would contemplate the same, Bosley would only say that all options are open.
Editas’s backers have made sure it has a war chest to engage in both technological and IP battles. It was launched in November 2013 by three of the Boston area’s biotech startup creators, Polaris, Third Rock Ventures, and Flagship Ventures (along with Partners Innovation Fund) with a big $43 million Series A round, and touted contributions from five of the most prominent researchers in the field: Feng Zhang (the Broad Institute); George Church (Harvard University); Jennifer Doudna (University of California, Berkeley); David Liu (Harvard); and Keith Joung (Massachusetts General Hospital).
Bosley is one of the more prominent names in Boston biotech, coming up through the ranks on the business development and venture sides of the field via stints at Biogen Idec (NASDAQ: BIIB), Highland Capital Partners, Alkermes (NASDAQ: ALKS), and Adnexus Therapeutics. Most famously, she led Bedford, MA-based Avila Therapeutics, a developer of covalent cancer drugs started up in 2007, and sold to Celgene for $350 million up front (and up to $925 million overall) five years later.
Since Avila was acquired, Bosley has kept busy. She became an entrepreneur-in-residence at the Broad Institute, chaired the board of Cambridge-based vaccine developer Genocea Biosciences (NASDAQ: GNCA), and took on board seats at new startups like Scholar Rock and more developed biotechs like Galapagos and Coco Therapeutics.