In the wake of big patent decision, it’s business as usual for CRISPR therapy developers

NEW YORK — Earlier this month, the U.S. Patent and Trademark Office ruled that CRISPR patents key to developing human therapies belong to the Broad Institute of MIT and Harvard, ending the latest chapter in a bitter seven-year battle between the Broad and the home institutions of Jennifer Doudna and Emmanuelle Charpentier — the two scientists who won the Nobel Prize for creating the revolutionary gene-editing technology.

But for all the acrimony exchanged and millions of dollars of legal fees spent by the academic institutions where CRISPR was first invented, the companies that are actually turning the technology into medicines are plowing through the fallout of the decision with little more than a collective shrug.

“I think it’s really a topic for people interested in IP law,” Lawrence Klein, the chief operating officer of CRISPR Therapeutics said Thursday during the 2022 STAT Breakthrough Science Summit in New York City.

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Klein said the ruling isn’t changing the company’s focus or its commercialization strategy. “At the end of the day, there might be a modest economic impact one way or another, but when you zoom out there’s so much exciting science and innovation going on that’s going to improve people’s lives,” he said. “It’s just not something to focus on, in my opinion.”

Laura Sepp-Lorenzino, executive vice president and chief scientific officer at Intellia Therapeutics, was quick to concur. “Fully agree,” she said.

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To develop their pipelines of gene editing treatments, Intellia and CRISPR Therapeutics both licensed patents from the University of California. CRISPR Therapeutics has had early clinical success treating inherited blood conditions, and Intellia recently announced that a one-time treatment of its therapy for a genetic nerve disorder lowered levels of the disease-causing protein for up to nine months — the first biotech company to report success with a so-called in vivo CRISPR-based medicine.

Editas Medicine, which for now holds a competitive intellectual-property advantage with its exclusive license on applications of Broad scientist Feng Zhang’s patents for treating human disease, has fallen behind its rivals in the push to market. After a series of setbacks, a clinical trial of its treatment for genetic blindness finally began last year, but so far has shown mixed results.

As things stand now, Editas could, in theory, seek to block competitors from selling CRISPR-based medicines, if and when they receive Food and Drug Administration approval to do so. Such a scenario is rare, but it has happened before. In 2014, Amgen sued its partners Sanofi and Regeneron over alleged patent infringement on its PCSK9 inhibitor, Repatha, successfully scuttling the drug’s launch in the U.S. market. But far more routine is for companies to sort out licensing deals that benefit them both.

On Thursday, Editas leadership seemed to indicate, more strongly than it has in previous public statements, that the latter is the likely way this will play out for CRISPR.

“It is never going to be our intention to prevent any of the other gene editing companies from bringing a therapy to the market,” said Mark Shearman, Editas executive vice president and chief scientific officer. “I’m sure at some point in the future the legal teams across the organizations will figure this out.”

Still, much remains up in the air, even as the intellectual property around CRISPR is growing increasingly complex. Patent offices in other countries have reached different decisions about who invented what. And additional parties have entered the fray — ToolGen in South Korea and Sigma-Aldrich, owned by Merck KGaA in Germany, are challenging the ownership of some of the early, foundational CRISPR patents, legal disputes that could take years to play out while further embroiling both the Broad and the University of California, and the companies that licensed their gene-editing technology.

Decisions down the road could shift the landscape yet again. And meanwhile, no one wants to slow down the push toward developing what could be one-time cures for long-neglected diseases.

“I think we’ll be talking around the table,” Sepp-Lorenzino said, addressing Shearman to a ripple of timid laughter from the audience. “Because you need some of our patents, and who knows, maybe we need one of yours. Or maybe not, so we’ll see.”

Source: STAT