Gene-Editing Company Editas’s Ambitions Rest on Shaky Foundations

Gene-editing firm Editas today became one of the first companies to go public this year, raising $94 million that it hopes to use to develop a technology called CRIPSR-Cas9, which can make very precise changes to DNA, into a powerful new tool for treating illness.

The move, while expected, is bold for two reasons. First, no such therapy has been approved for clinical use in either the U.S. or Europe.

More importantly, Editas doesn’t even know for sure whether it even has the right to use CRISPR. Two of the scientists who founded the company (Jennifer Doudna of the University of California, Berkeley, and Feng Zhang of the Broad Institute and MIT) each claim to have invented CRISPR independently. They are now tangled in a complex legal dispute. Currently Zhang holds the upper hand, as he has been granted a dozen key patents by the U.S. Patent and Trademark Office, and Editas licenses them from Zhang and the Broad Institute. But Doudna contests Zhang’s primacy, and if she wins, Editas could be in trouble.

Even if the company overcomes these hurdles, success is far from guaranteed. CRISPR is still a nascent technology, but Editas already faces competition from two other startups, Intellia Therapeutics and CRISPR Therapeutics. The latter recently announced a $300 million joint venture with Bayer AG to develop new drugs for a range of illnesses.

What’s more, ushering a new drug or therapy through the arduous process of clinical trials and regulatory approval is time-consuming and expensive. Editas CEO Katrine Bosley says the company could begin its first clinical trial by 2017, but CRISPR has never yet been used to edit the DNA of a person. If the company is to see its plans through, it will almost certainly need much more money.

(Source: Wall Street Journal)