No politics this time!
Unless science politics counts.
DESIGNER BABIES ARE OK SOMETIMES
The National Academy of Sciences, bless its heart, has tackled once again the genomic future that is just a moment away from being our genomic present. Last time it was a sane report on GMOs. This time it’s a sane report on prospects for editing the human genome. This includes even making disease-related changes that can be inherited–but a firm “NO” on designer-enhanced babies except for a select few diseases. (The PDF download is free here.)
At Speaking of Science, Joel Aschenbach says the report is a “flashing red light for CRISPR,” the highest-profile gene editing technique. I’d characterize it as a flashing yellow light, or nearly so, at least by comparison with the position an NAS-sponsored group took in December 2015. At that time, the recommendation was strongly against germline modification for any reason, meaning changing gene(s) of human eggs/sperm/embryos with the intention of starting a pregnancy. These are modifications that could be inherited by future generations.
The new report declares, Aschenbach says, “if and when such interventions are proved safe — which could be in the near future — and if numerous criteria are met to ensure that such gene editing is regulated and limited, it could potentially be used to treat rare, serious diseases.” No enhancements allowed, no designer babies with greater intelligence or stronger musculature.
Diseases already under investigation with gene-editing techniques include “sickle cell disease, hemophilia, cystic fibrosis, Duchenne muscular dystrophy, genetic forms of blindness, and, of course, cancer,” acccording to Ryan Cross at C&EN. He points out that successful gene editing would not be a treatment, but a permanent fix. Cross’s post is a nice overview of the stunning pace at which CRISPR has taken over biology, and the technical problems remaining to be solved.
“The panel of experts also took into consideration the reality that genome editing will occur around the world regardless of an endorsement. Therefore, the committee saw it as beneficial to put forth stringent guidelines for how to do it responsibly,” Beth Mole notes at Ars Technica.
NIH Director Francis Collins recently blogged about the prospects for the new genome-editing methods–he calls it find-and-replace DNA editing–as a much more efficient tool for gene therapy, which seeks to replace a malfunctioning gene with one that works. That field has struggled with technical barriers and bad press for decades.
“Instead of threading the needle through the cell membrane with a bulky gene, researchers are starting to design ways to apply these tools in the nucleus—to edit out the disease-causing error in a gene and allow it to work correctly,” he says. They can already report some success (in mice) with CRISPR for curing a rare inherited human immunodeficiency called chronic granulomatous disease (CGD).
Stem cell researcher Paul Knoepfler is not terribly happy with the new report, in part because he wants a moratorium on germline modification. He concedes that a moratorium would present problems of its own, for example enforcement. Still, “The report in some places does not use strong enough language to counter the idea of human germline modification,” he says.
THE CRISPR PATENT HAS BEEN AWARDED. BUT WHO’S THE WINNER?
The day after the genome-editing report from the National Academies, it just about disappeared from media attention. That’s because, boom, came a somewhat more arcane declaration that’s quite a bombshell for the genome editing world: the US Patent Office has awarded patents on the premiere genome-editing method, CRISPR-Cas9, to the Broad Institute of Harvard and MIT. STAT’s Sharon Begley tells all.
This award despite the fact that science itself clearly believes that CRISPR’S originators are Jennifer Doudna of the University of California – Berkeley and Emmanuelle Charpentier, now of the Max Planck Institute for Infection Biology, Berlin. The two have been awarded major science prizes for the work, amounting to millions of dollars.
Broad’s Feng Zhang later showed the method could work in cells. The patent ruling means Broad’s work is different from the University of California’s. At Nature News, Heidi Ledford says the decision means that Broad will control what are likely to be the most lucrative applications of CRISPR-Cas9.
But that’s not entirely clear. As the hed on Nidhi Subbaraman’s post at BuzzFeed read, “A Nasty DNA Patent Fight Just Ended But Experts Are Still Confused“.
Media accounts were certainly muddled. Some outlets treated the decision as a knockout for Broad. At Fortune, Clifton Leaf compared the patent decision to the 1973 Frazier-Foreman boxing match. In the biggest biotech fight of the century, he said, “Down goes Doudna!”
But the University of California has by no means given up. For one thing, it might appeal the decision.
Doudna pointed out to TechCrunch that, even though the Patent Office did not throw out the Broad Institute’s claims, it also did not throw out her claim either. “We’re actually anticipating getting our patent issued finally, one that has very broad claims,” she said. “The analogy I’ve used to explain this is the Broad Institute’s patent is for green tennis balls but the patent we will have is for all tennis balls.”
Ledford notes that the two groups might end up settling. It’s also possible that companies who want to use the techniques will have to buy licenses from both Broad and California, driving up research costs.
The scientific impact of genome editing, especially CRISPR, is hard to overstate. So the patent ruling is commensurately huge. It will bring in tons of money, probably govern who gets annointed by science history, and could determine the fates of dozens of biotech startups. Antonio Regalado of MIT’s Technology Review has details, especially financial ones.
In a STAT post describing winners and losers in the CRISPR patent decision, Meghana Keshavan declared the Nobel Prize committee a winner. “It’ll be fun to decide who, precisely, merits the inevitable CRISPR Nobel. The winner of the seminal patent doesn’t necessarily win the award, so the vaunted prize committee will get to make its own judgment call. And it’ll be closely watched. Historically, this particular institution tends to neglect scientists who are female. The CRISPR prize could be a chance to right that wrong.”