The Patent Trial and Appeal Board (PTAB) has ruled in favor of the Broad Institute of MIT and Harvard in its dispute with the University of California (UC) Berkeley and the University of Vienna over intellectual property issues surrounding CRISPR/Cas9 technology. The powerful technology can be used to edit and modify the genome of organisms.

The saga began with the discovery of CRISPR/Cas9 in 2012. Dr. Jennifer Doudna, a structural biologist at UC Berkeley, in collaboration with Dr. Emmanuelle Charpentier, now director of the Max Planck Institute for Infection Biology, developed the technology for use with prokaryotes, or bacteria cells, and has a broad patent for their initial invention. Shortly thereafter, however, Broad’s Dr. Feng Zhang determined how to use the technology with eukaryotic cells, or plant and animal cells, and obtained several patents protecting this discovery. The UC Berkeley challenged these patents, claiming that Broad had simply built on its earlier discoveries.

The PTAB saw it differently. It sided with Broad, agreeing that the institute had indeed designed a new system. To date, 50 patents with claims to CRISPR/Cas9 have been issued, and 13 of those belong to the Broad Institute, MIT and affiliated groups.

With the interference removed, UC Berkeley is moving forward with its patent claim for use of the technology with eukaryotic cells. But for now, Broad is in the driver’s seat.

“This decision is a clear victory for Broad,” said Jacob Sherkow, an intellectual property attorney at the New York Law School in New York City. “My guess is that UC Berkeley will appeal it to the U.S. Court of Appeals for the Federal Circuit, with the hope of winning outright. If I was their lawyer, that’s what I would recommend.”

For academic institutions, which conduct research for noncommercial purposes, this decision may have little impact. Both institutions have been generous in licensing the technology to academic institutions both in the U.S. and abroad.

In the commercial world, however, the situation is different, in part because of the approach UC Berkeley and Broad have taken in licensing their intellectual property. They have chosen to license them to surrogate companies, which take on the patent, the profits and much of the risk. UC Berkeley has granted an exclusive license to conduct research on the whole genome to a company called Caribou Biosciences, which in turn has a sublicense agreement with Intellia Therapeutics. This company is working on in vivo indications for liver disease and ex vivo systems with stem cells. The Broad Institute has a exclusive licensing agreement with Editas Medicine, which is also working on human applications.

For now, companies like Intellia, which has its license from UC Berkeley, are putting a positive spin on the ruling. Though admitting to being disappointed in PTAB’s decision, Jose Rivera, executive vice president, General Counsel & Operations of Intellia, said, “It is important to note what the ruling did and did not do. It did not make a determination about who invented the technology first, and it acknowledged the overlap between the two sets of claims. With the interference removed, UC Berkeley’s patent can move forward, and we believe they will prevail.”

But Sherkow has a different perspective. “It is unclear how generous Broad is going to be in giving licenses to technology companies,” he said. “What’s more, it is possible that if UC Berkeley receives its patent, companies may have to pay fees to both institutions to use the CRISPR/Cas9 technology. Either way, it is likely that they will be paying fees to Broad.”

If that’s the case, the results of a survey of CRISPR trends may be providing even more good news. Conducted by Synthego, a company that provides synthetic RNA and other genome-editing tools, the survey was designed to find out directly from the research community how scientists are using CRISPR technology and what their plans are for the future.

The survey found that 87% of users are new to gene editing, and 55% of that group have plans to conduct experiments within six months. The survey also showed that 35% of new researchers plan to conduct experiments directly related to therapeutic applications, with stem cell research representing 18%, drug discovery 11% and CAR-T therapies 6%.
“It was a big surprise to us that the large majority of new CRISPR users are also new to gene editing,” said Mike Dabrowski, president and co-founder of Synthego. “This tells us there’s a tremendous hands-on interest in CRISPR as a genome editing technique. We were less surprised to hear that scientists were interested in using CRISPR for therapeutics, as well as with an incredibly diverse set of organisms.”

The Synthego survey points to the potential for new customers, who may find that Broad is a force to contend with. If Sherkow’s predictions prove to be correct, these customers may be paying licensing fees to Broad at some point down the road.

To get around Broad’s potential influence, academic institutions may want to reconsider exclusive licenses, which may not be necessary. “Instead of giving companies licenses to work on the entire DNA of the human genome, they may want to consider only part of it or for specific indications,” Sherkow noted. “This also may be a good time to look for new nuclei and recombinant nuclei that can be used in the system.”

Sherkow then offered one more suggestion. “Before the appeal process begins, companies may get a better deal from Broad. Perhaps they should start thinking about making nice with the institute.”  

This article was reprinted from Volume 21, Issue 08, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »