Last week a panel of judges at the U.S. Patent and Trademark Office in Alexandria, Virginia, heard arguments as to who should own the rights to the century’s biggest biotechnology invention to date, a precise gene-editing system called CRISPR-Cas9 that has the potential to treat serious human genetic disorders and create designer crops that resist drought and pathogens.
Embroiled in the dispute are the Broad Institute of MIT and Harvard, which holds 13 CRISPR-related patents, and the University of California, Berkeley, which believes it is the true inventor of the technology. Groups at the two universities are fighting for ownership of CRISPR gene editing in eukaryotic cells (those of humans, plants, and animals), which represents the most lucrative uses of the technology.
At stake are billions of dollars tied up in numerous commercial agreements with biomedical and agricultural companies. The outcome of the so-called patent interference could render some of those contracts invalid. But the patent judges’ decision—expected in early 2017—is not likely to put any CRISPR companies out of business or even slow the lightning pace of research and development in commercial laboratories, experts say.
“The success or failure of any company is not determined by patents alone,” says Mark Shtilerman, an intellectual-property lawyer at Deerfield Management, which has invested $20 million in Editas Medicine. Rather, he says, a company’s pipeline is more important.
While Editas has exclusive licensing rights to use CRISPR technology from the Broad Institute to make medical treatments, other companies, including Intellia Therapeutics, CRISPR Therapeutics, and Caribou Biosciences, hold licenses or sublicenses to the rival intellectual property controlled by the University of California and several European inventors. Even with the fate of key patents up in the air, these companies have attracted a combined total of more than $1 billion in venture capital and are racing to develop therapeutics that use DNA editing to correct disease-causing genetic alterations.
Editas, Intellia, and CRISPR Therapeutics declined to comment for this story.
If the patent judges decide that the Broad is the official inventor of CRISPR and upholds all its patents, it’s likely that most other companies would then need to license the technology from the Broad or Editas, since these patents are fundamental to using CRISPR in eukaryotic cells, Shtilerman says. But it’s possible the judges could rule in favor of the University of California, in which case Editas and other companies aligned with the Broad would have to negotiate new license agreements.
Harvard genetics professor George Church, a CRISPR researcher who is also a founding member of Editas, says he hopes that if the Broad wins, Editas will grant what is known as a sublicense to other companies developing CRISPR-related biotech drugs so they can “get on with their work.” He says he would be surprised if the winner of the patent battle didn’t dole out such licenses.
“I don’t see the point in having winners and losers,” Church says. The more companies working on this technology, the greater the chance for one of them to develop a blockbuster drug, he says.
In exchange for a sublicense, companies would agree to share a certain portion of profits with the patent holder.
In the agricultural sector, DuPont has licensed CRISPR technology from Caribou Biosciences, and Monsanto has licensed patents from the Broad Institute. DuPont is already working to commercialize a CRISPR-edited corn product that it says will be available in five years.
Neal Gutterson, vice president of research and development at DuPont Pioneer, said in a statement that the company does not speculate on ongoing legal proceedings. But he acknowledged that DuPont “has a strategy in place to position our business as a leader in the application of CRISPR-Cas in agriculture.”
Colleen Tracy James, an intellectual-property lawyer specializing in life sciences at the firm Mayer Brown, says it could take as little as a few weeks for companies to negotiate and get a new license from the official inventor, if needed. She says the winner “has an incentive to do it quickly and get the revenue.”
A third possible outcome of the patent hearing is that the judges could award patent rights to both the Broad Institute and the University of California. In that case, the companies licensing CRISPR technology would need to determine which institution owns the rights to the specific application they are using.
Until then, companies developing potentially life-saving drugs are legally protected under what’s known as a “safe harbor” exemption, Shtilerman says. The exemption means that companies can conduct research using a patented invention even if they don’t hold a license to use that technology.