THE BLOG
12/10/2014 04:56 pm ET Updated Feb 09, 2015

CRISPR Opportunities ... For What? And for Whom?

Money and deals are flowing into companies that promise to edit genes. Human, animal, plant, all kinds of DNA may be on the cutting board. And once the replacements are pasted in, the results could dramatically change our lives -- for better or for worse.

In the meantime, scientists and lawyers and venture capitalists are jockeying for position to see which companies will come out on top. The rest of us should pay attention; it's our world they are looking to remake.

This burgeoning industry is founded on CRISPR technology, which promises to enable the precise editing of genomes. CRISPR/Cas9 stands for "clustered, regularly interspaced, short palindromic repeat" and an associated protein (not always mentioned). A brief video introduction from UC Berkeley's Innovative Genomics Initiative (IGI) gives an overview of the editing process. A webinar put on by Genetic Engineering & Biotechnology News, featuring Harvard professors George Church and Feng Zhang, gives more detail.

The first prominent company intending to exploit this technology was Editas, which was founded by five of the leading scientists in the field, with $43 million in capital. Editas recently announced licensing deals with Massachusetts General Hospital, Duke University, Harvard University and the Broad Institute, all for intellectual property related to the use of CRISPR/Cas9 as well as other genome-editing technologies.

Bio-It World has more about the implications of these deals for prospects of both ex vivo and in vivo drug development, and quotes CEO Katrine Bosley as saying they are "at least a couple years from the clinic." In a press release, Bosley said:

"We all share the goal of translating this cutting-edge science into breakthrough medicines for people with genetically-driven diseases."

Generally, Editas is looking to create "robust medicines" that will enable "the prevention and treatment of human or animal disease, and broad agricultural use."

This superficially bland ambition has some worrying overtones. One major concern is the apparent assumption that genomics can reliably predict disease in time to prevent it, which is only true of a small number of genetic disorders. Yet some of those most deeply involved in the research are given to ambitious speculation about, for instance, engineering people -- to be virus-proof (though in the process they'd be unable to reproduce with present humans), or to have enhanced traits of various kinds, which would also be inheritable. The implications of this are profound.

One of the five co-founders of Editas, which is based in Cambridge, MA, was Jennifer Doudna, the UC Berkeley professor who first developed CRISPR technology, working with Emmanuelle Charpentier, who is now based in Germany. The Boston Globe suggests that they are "shoo-ins for a Nobel Prize," and in November they each won a "Breakthrough Award" worth $3 million, from various Silicon Valley billionaires including Yuri Milner, Mark Zuckerberg, Sergei Brin and others. However, Doudna is no longer listed as one of the company's Scientific Advisors.

Commercially, Doudna seems to be focused on Berkeley-based Caribou Biosciences, whose motto is "engineering any genome, at any site, in any way." Charpentier co-founded CRISPR Therapeutics, with offices in Basel and London, financed by Versant Ventures and focused on "developing cures for human genetic diseases."

Caribou recently announced the co-founding, with Atlas Venture, of Intellia Therapeutics, which is based in Cambridge, MA. Intellia has raised $15 million [pdf] in a financing round led by Atlas Venture and Novartis, and seems to be positioning itself as providing a drug development platform. Caribou will also "apply its technologies toward the development of new therapeutics in the anti-microbial and animal health spaces."

There seems to be some serious jockeying for position here. The patent situation may become complex -- Broad has been awarded one important patent, but other applications remain on file, and appeals might overturn the Broad decision. (See this article from MIT Technology Review.) Quite how the business side shakes out is hard to predict.

Meanwhile the general hype continues: Scientific American calls CRISPR the top "world-changing idea" for 2014, and is asking [mostly behind a paywall]:

Is the Gene-Editing Revolution Finally Here?

MIT Technology Review [free registration required] named CRISPR the #1 "breakthrough technology" of the year. (The "smartest company" title they gave to Illumina, the genome sequencing company.)

But what, exactly, are these companies going to do with the technology? Will it involve germline -- that is, inheritable -- alterations of the human genome? Doudna is "uncomfortable" with that idea, and has acknowledged that there is a "dark side," especially if the technique were to be "used for trivial or even harmful uses." But that's about as much caution as you'll find among these enthusiasts.

For a partial corrective, see a new paper by Motoko Araki and Tetsuya Ishii on the "International regulatory landscape and integration of corrective genome editing into in vitro fertilization." They survey the status of genome editing, note how it could be integrated with IVF and "address some ethical and social issues that would be raised when each country considers whether genome editing-mediated germline gene correction for preventive medicine should be permitted."

As investment and futuristic visions rush ahead, understanding and awareness of what's at stake are trailing. A biopolitical awakening is urgently on the agenda.