BioPharma

A silver lining in the CRISPR court case

On Tuesday, judges heard verbal arguments from both sides of the CRISPR intellectual property fight, a technology that could prove to be the greatest biotech advance in recent history. But the outcome matters less than what is happening in the field right now.

Gene editing, CRISPR

It’s a case that will go down in history: The Broad Institute Inc v. Regents of the University of California, patent interference No. 106,048.

On Tuesday, both sides made what are essentially closing remarks, appealing to a panel of three judges at the Patent Trial and Appeal Board (PTAB) in Alexandria, Virginia. At stake are licensing rights to what is arguably the most disruptive biotech advance in recent history, CRISPR.

Robert Underwood, a partner at McDermott, Will & Emery, has been following the story closely. In a phone interview during the interference proceeding, he discussed the legal process and the potential ramifications for biotech innovation.

“It is a technology that by all accounts has tremendous potential to influence the human condition,” Underwood said.

For this reason, he believes the real story of CRISPR is taking place outside the proceedings room — and it’s good news.

Throughout the world, academic and industry scientists continue to advance the technology. Multiple companies have launched head-first into tools and therapeutics that fundamentally rest on CRISPR inventions. And investors have backed them, despite the open-ended patent case.

“There’s this cloud over who is going to own it, what the ownership situation is going to look like, what the licensing situation is going to look like,” Underwood said. “I think the encouraging part is that sort of uncertainty does not seem to have had a major impact on slowing the development of the technology and the application of the technology.”

Already, Berkely, California-based Caribou Biosciences has raised $44.46 million for its CRISPR program. Intellia Therapeutics went public in just two years, raising $108 million in its IPO. The eponymous CRISPR Therapeutics raised $127 million in private funding, then also went public on Oct. 19, 2016.

To date, Editas Medicine has earned the greatest backing. It was founded in 2013 by Feng Zhang and Jennifer Doudna, the two competing inventors of CRISPR gene editing.

Doudna left the company when the now-contested patent was awarded to Zhang and his team at the Broad Insitute and Harvard University. Editas quickly signed exclusive rights to the IP and later licensed other CRISPR technology from Massachusetts General Hospital and Duke University.

Combined, Editas raised $210 million in three rounds of private investment, before going public on Feb. 3, 2016. All in the face of uncertainty over its foundational patent.

Outside of therapeutics, CRISPR unleashes countless new possibilities as a scientific tool. In simplified terms, it allows scientists to edit an organism’s genome, from knocking-out genes in mouse models to modifying agricultural crops and much, much more.

In this setting, academic researchers are free to use the technology, though they would need to license rights from the official inventor to commercialize any ideas.

In a 2014 interview, Zhang told Bio-IT World that he felt it was important to keep the technology open for the wider scientific field.

“Something like CRISPR is a foundation tool,” Zhang said. “I think making sure these foundation tools are open is important… What my lab has done is try to make the information, the technical know-how, as well as the physical reagents, as accessible to everybody as possible.”

Whether he will have final say over its fate remains unknown. The USPTO ruling is likely months away — Underwood estimates it will be delivered in February. If Doudna is deemed to have a case, more deliberation may be needed.

The patent dispute centers around the progression of the science. Doudna’s lab at UC Berkely undoubtedly filed its patent first. Zhang’s patent was submitted months later, yet it made an important leap in the application of the technology.

Zhang’s filing described the use of CRISPR in eukaryotic cells; plants, animals, and humans, not just simple organisms. It is that advanced application that has kicked-off a billion-dollar field of biotech research.

The University of California’s argument says the transition from simple organisms to eukaryotes was a no-brainer; a simple evolution of Doudna’s work, not a separate technology.

Due to the patent overlaps and the timing of the two filings, the USPTO declared what is known as an interference proceeding. It is applied when two inventors file a patent application for what is considered to be the same invention within 6-12 months of each other. If the patent office deems both applications worthy of a patent, an interference can be declared.

It’s an outdated U.S., patent regulation, Underwood said, which was replaced with a ‘first-to-file’ system on March 6, 2013. All patents filed before that date remain subject to the interference laws.

“The ultimate goal of that proceeding is to determine who was the first to invent the subject matter,” Underwood stated. “The first to invent is entitled to the patent. The second to invent is not.”

So what of the companies that have licensed Zhang’s CRISPR technology from Broad and Harvard? If the judges determine Zhang is the rightful owner of the invention, those licensing deals will remain intact.

On the other hand, if the Doudna’s side wins, the companies would likely need to negotiate the same agreements from the University of California. Though they might be a little out-of-pocket, the critical part is that the field keeps moving forward.

A third possibility is that the judges, who have both biotech and legal training, will decide there is distinct merit in both patents. The companies licensing the technology will then need to determine who has what rights to their specific application.

“We’ve seen these types of disputes before,” Underwood noted. “I think that what makes this one perhaps a little bit unique is that the technology has such, such high promise that it’s really getting the attention of everybody.”

There are hundreds of millions — if not billions — of dollars on the line. But the potential value CRISPR could have conquering human diseases? Priceless. 

Photo: wildpixel, Getty Images

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