The fight over who owns the most promising technique for editing genes—cutting and pasting the stuff of life to cure disease and advance scientific knowledge—has been a rough one. A team on the West Coast, at UC Berkeley, filed patents on the method, Crispr-Cas9; a team on the East Coast, based at MIT and the Broad Institute, filed their own patents in 2014 after Berkeley’s, but got them granted first. The Berkeley group contended that this constituted “interference,” and that Berkeley deserved the patent.
At stake: millions, maybe billions of dollars in biotech money and licensing fees, the future of medicine, the future of bioscience. Not nothing. Who will benefit depends on who owns the patents.
On Wednesday, the US Patent Trial and Appeal Board kind of, sort of, almost began to answer that question. Berkeley will get the patent for using the system called Crispr-Cas9 in any living cell, from bacteria to blue whales. Broad/MIT gets the patent in eukaryotic cells, which is to say, plants and animals.
Its confusing. The patent that the Broad received is for the use of Crispr gene-editing technology in eukaryotic cells. The patent for the University of California is for all cells, saysJennifer Doudna, the UC geneticist and co-founder of Caribou Biosciences who co-invented Crispr, on a conference call. Her metaphor: They have a patent on green tennis balls; we have a patent for all tennis balls.
Observers didn’t quite buy that topspin. If Caribou is playing tennis, its looking like Broad/MIT is Serena Williams.
UC does not necessarily lose everything, but they’re no doubt spinning the story, saysRobert Cook-Deegan, an expert in genetic policy at Arizona State Universitys School for the Future of Innovation in Society. UC’s claims to eukaryotic uses of Crispr-Cas9 will not be granted in the form they sought. That’s a big deal, and UC was the big loser.
They have a patent on green tennis balls; we have a patent for all tennis balls.Jennifer Doudna, UC Berkeley
UC officials said Wednesday that they are studying the 51-page decision and considering whether to appeal. That leaves members of the biotechnology sector wondering who they will have to pay to use Crispr as part of a business—and scientists hoping the outcome wont somehow keep them from continuing their research.
Why It Matters Who Wins
Someone is going to make a lot of money licensing Crispr. And someone is going to make lot of money on therapies based on Crispr. Thats why, the day before the decision, the National Academy of Sciences released a long document laying out what kind of Crispr-based human therapies were kosher—so no one goes the full Gattaca.
In fact, the moneymaking part has already begun. Startups are getting funding based on Crispr-based business plans. Editas Medicine, which licenses the Broad patents to work on treatments for genetic disorders in human beings, had a 30 percent stock bump on word of the patent decision. It certainly caused some concerns, because depending on how the courts were going to rule on the two claims, if you went with one, you could lose, right? says Edison Liu, CEO of the Jackson Laboratory, a major source of genetically modified mice used in research. Jackson Labs has licenses from both sides, and since it aims at academic uses, gets better terms than a Silicon Valley biotech startup might.
But not everyone can make multiple deals. Its a bit frustrating that the patent office has done it this way, says Eric Rhodes, CEO of ERS Genomics, which licenses UCs Crispr technology to other companies for non-human therapeutic uses. A lot of people were hoping they would make a decision to go with one group or another.
Commercial outfits hoping to make new therapies will, for now, have to pay both institutions big fees. ERS Genomics, for example, charges from $10,000 to small start-ups to $1 million to large pharmaceutical firms. And an attenuated patent dispute could mean more red tape and lawyers on retainer for biotech scientists.
On the plus side, the patent fight doesnt look likely to slow basic research, or even research with an eye toward commercialization. Neither UC nor Broad will charge academic scientists who want to use Crispr just to better understand plant diseases, lets say. I dont think it’s going to slow down therapeutic development, says Rhodes.
The same goes for people trying to use animal models like mice. Thats good, because the models and the technique are ubiquitous. If youre creating a mouse model, you just have to use this. Its a little bit like, once internal combustion with a carbon source came out, nobody was spending a lot of money on the steam engine, you know? says Liu.
Two Biotech Institutions Enter; One Biotech Institution Leaves
The final outcome of this patent fight may be years away—especially if Berkeley appeals the decision. Its one battle in a larger war, says Jason Sherkow, a bioscience patent law expert at New York Law School. But its a very big, important battle.
There will be paperwork and cross-licensing that will have to be worked out before drugs are commercialized. Hopefully some of this patent situation will work itself out before then.Eric Rhodes, CEO, ERS Genomics
Thats because if a biotech company develops a megahit drug or treatment using Crispr, itmight now have to pay the Broad megabucks in fees. “There will be paperwork and cross-licensing that will have to be worked out before drugs are commercialized,” says Rhodes. “Hopefully some of this patent situation will work itself out before then.” Broad/MIT might even go Hollywood here, taking a small upfront fee in return for points off the gross revenue down the road. It does paint a dark and foggy picture for any of the companies that received licenses that originally came from Berkeley, says Sherkow.
Here’s where things get a little sketchy even for the most basic of basic researchers—because you never know, exactly, what youre going to come up with. If you are contemplating creating something that would be marketed in any way, Liu says, then it is actually the commercial end of it, the sale of the product, that will probably require a license.
This isn’t the first time universities and companies have fought over a lucrative invention that could further scientific research. Harvard fought for years for rights to the cancer-prone, genetically engineered oncomouse. Until the US Supreme Court took them away in 2013, Myriad Genetics held the rights to genes for breast cancer. In 1980, researchers at UC San Francisco and Stanford figured out how to make recombinant DNA in bacteria, and patented it. In that last case, university licensing offices negotiated ways to let people use the technology to do science and create businesses. But the time for that kind of sensible negotiation seems to have passed with Crispr. Its the only thing all parties really agree on: Too much is at stake.
