Image

Saturday, October 22, 2016

Editing a blazing trail into a heated trial - daily life in the world of CRISPR!


Logo      Image result for editas logo     Image result for Intellia logo

Science and biotechnology continue to push ahead exponentially, and in today's world that means making big bets ahead of truly understanding both the full capacity and potential dangers inherent to certain technologies. It's hard to think of a hotter yet simultaneously controversial example than CRISPR, the next generation gene-editing tool that is capturing not only the imagination of us scientists, but even those in Hollywood!


Yes, believe it or not, even before CRISPR has gotten close to the nail-biting moment of using it on humans, Jenny-from-the-block herself, Jennifer Lopez, apparently wants to produce a TV show centred around the nascent technology. That's quite something for an untested, unproven therapy, and it probably does reflect not only that hardcore science is becoming more and more mainstream these days, but also that CRISPR itself is causing considerable controversy all on its own. I just can't wait to hear the words "clustered regularly interspaced short palindromic repeats" on a TV show; bet you can't say that fast, three times in a row, Jen! 

The potential of CRISPR to change the world of medicine in the post-genomics age is scary-level huge, and of course that potential is not limited to classical medicine but in effect also brings us crashing into a brave (frightening) new world where science can be used to edit something which has evolved over an eternity - our DNA - and messing with that in anything but the most controlled of conditions could have devastating consequences. 

But this is the world we are living in today, and the relentless push of next-generation therapeutic approaches such as CAR-T and CRISPR will continue to revolutionise that world, and only a lack of money could hinder or prevent that. This does not seem to be an issue thus far for the "big three" in CRISPR commercialisation who have now all gone public in recent IPOs; that trio of course consists of Editas Medicine, Intellia Therapeutics and CRISPR Therapeutics. 

Editas ($EDIT) was the first out of the gate back in February, with Intellia ($NTLA) following a few months later, and this week it was the turn of CRISPR ($CRSP). The former two raised about $100M each (on top of considerable VC and pharma money) in their respective IPOs, while CRISPR came in at around $56M, failing to hit their initial target of $90M. Analysts were quick to jump on this, implying that the bottom is already falling out of the market, and this fact had negatively impacted $CRSP's raise. 

While investors may indeed be a little less sanguine on this avant garde and risky bet than a year ago, it is worth pointing out that both $EDIT and $NTLA have seen their market cap nibbled at and all three companies actually have a similar valuation today. In fact, $CRSP is pretty much par for the course, with a current valuation just short of the $500M mark, which is in the same ballpark as the other two after their valuations slipped somewhat and were adjusted post-IPO. 

The confounding factor here is not only which one of these three will get to the first-in-man clinical trial, first, but they are also all facing the reality of the ongoing and explosively contentious patent wars regarding who-owns-what with respect to CRISPR technology. At the heart of the dispute is whether it is indeed UC Berkeley and Doudna/Charpentier who should own key patents in the field, or whether the current status quo should be upheld such that the great majority of the key patents at stake should remain the property of the Broad Institute and Feng Zhang. Additionally, although it garners way less media attention, another prestigious institution, the Rockefeller, is also involved in the fracas and they are claiming that one of their scientists (Marraffini) should be named on specific Zhang/Broad granted patents. 

Apart from egos and bragging rights, there is a vast amount at stake in this patent interference case, and the decision of the USPTO could have staggering consequences for those who do not prevail. At the very least, their bottom line will be affected by being forced to pay no doubt elaborate license fees to use the technology therapeutically. Or, if a decision was made against Zhang, the new owners could license the technology to a competitor of Editas, for example, and the company would be in very deep you-know-what. At worst, if investor and pharma attention swings away from the losers to focus on the (new) owners of the IP, the other companies may struggle to even survive. The temperature during the legal exchanges and between the major scientific players must be a little crisp, to say the least! 

I am in Toronto next week for the Canadian "Gairdner Awards" lectures at the University of Toronto, and you can be sure I will be watching closely as Jennifer Doudna, Emmanuelle Charpentier, and the wunderkinde of the bunch, Feng Zhang all present their work. It will be interesting to see how they position their contributions, with all of the legal shenanigans and inter-institutional fighting still going on in the background. You can be sure I will be looking and listening for any signs of tension or bold claims of ownership during the presentations - watch this space for an update! 

No comments:

Post a Comment