Here’s a CRISPR first: Editas Medicine of Cambridge, MA, has filed paperwork for an IPO. Its S-1 document became public today, marking the first one from a company working to turn the gene editing system CRISPR-Cas9 into human therapeutics.
The reaction of the public markets to Editas, assuming it gets as far as an IPO, should be a fascinating event. The biotech bull market is receding farther from view, and public recognition of gene editing as a brave new world fraught with promises and perils is growing. What’s more, the fundamental ownership of the CRISPR-Cas9 technology is in dispute and headed toward a special type of patent fight that pits the patent holders (some of Editas’s founders) against their challengers, as I wrote about this morning.
All those factors, plus the company’s goal of bringing its first—and perhaps the first—CRISPR-Cas9 therapy to human testing in 2017, should add up to one of the biggest biotech stories of 2016.
While you ponder all that, here are eight facts gleaned from a first run through the Editas S-1.
—The company was incorporated under the name Gengine and (thankfully) changed its name to Editas in 2013.
—It accumulated losses of more than $75 million through September 2015. It also had $155 million in cash at that point.
—It intends to spend $15 to $20 million of its IPO cash on preclinical and clinical trials of its LCA10 program, to treat a rare genetic blindness, and up to $22 million on preclinical studies related to its collaboration with Juno Therapeutics (NASDAQ: JUNO).
–Beyond the LCA10 program, which CEO Katrine Bosley (pictured) said in November would be in the clinic in 2017, Editas will also aim to treat the eye disease Usher syndrome 2a and herpes simplex 1-related blindness, which is the most common infection-related blindness in the U.S.—35,000 new cases a year.
—While Editas has a key license to CRISPR-Cas9 technology from its cofounder Feng Zhang of the Broad Institute of MIT and Harvard—the subject of the patent dispute—it turns out the company does not have a license to the work of Zhang on the new protein, Cpf1, which Zhang and his collaborators said in September could be an improvement over Cas9 in the CRISPR gene editing system. According to the Editas document: “We do not have rights to Cpf1, and, if we were to seek such rights, there can be no assurance we could obtain such rights on commercially reasonable terms, or at all.”
—In addition to technology licensing fees, Editas will pay the Broad Institute and Harvard University up to $14.8 million collectively for every product approved in the U.S., E.U., or Japan, with additional sales milestones up to $54 million. The fees are lower if the products are approved for an ultra-orphan disease.
—The top shareholders are Flagship Ventures (16.6 percent), Third Rock Ventures, and Polaris Venture Partners (each with 15.6 percent). Bosley owns nearly as much Editas stock (4.8 percent) as institutional shareholders Viking Global, Fidelity, and Deerfield (all 5.7 percent). The Bill Gates-affiliated vehicle Bng0, owns nearly 9 percent.
—Editas’s core values are summarized by six words—Community, Resilience, Ingenuity, Science, Passion, Revolution—whose first letters spell CRISPR. Coincidence?