Editas licenses new gene-editing tool, raising stakes in drug development race

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The competition to use CRISPR gene-editing technology to develop medical treatments is hotter than ever.Shutterstock

Editas Medicine Inc. is licensing a new gene-editing system from the Broad Institute of MIT and Harvard, potentially giving the Cambridge biotech an advantage in the race to be the first company to market medical treatments based on the potentially revolutionary CRISPR technology.

In a new set of licensing deals disclosed Monday, three-year-old Editas said it obtained exclusive rights to develop therapies based on a genome-editing system called CRISPR-Cpf1, discovered by prominent Broad Institute scientist Feng Zhang and colleagues who have applied for patents based on the technology.

At the same time, Editas extended and broadened its existing licensing of the CRISPR-Cas9 editing system, which also is being licensed by several rivals from other academic institutions in the United States and Europe. The science is the subject of a long-running intellectual property dispute that is now before a three-judge panel of the US Patent Trial and Appeal Board.

Gene editing based on the CRISPR technology might be able to unlock cures for thousands of genetic diseases — and generate billions of dollars in revenue for drug makers that use it. The new editing system has generated excitement within the relatively small world of global scientists working on genome editing approaches.

While the CRISPR-Cas9 system cuts straight across the two strands of DNA — enabling researchers to revise, remove, and ultimately repair defects — the new CRISPR-Cpf1 system makes a staggered cut. That creates the potential for scientists to edit genes in a different way that might be more conducive to developing particular treatments, though it is too soon to say which diseases might be best targeted by either system.

"This is an incredibly efficient technology that brings a new gene-editing tool into our toolbox," Editas chief executive Katrine Bosley said. "Different types of mutations need different types of repair. This fits nicely with our existing technology. It gives us a really powerful and broad platform to make new therapies through gene-editing."

On Friday, four Editas competitors — including Cambridge's Intellia Therapeutics Inc., and Crispr Therapeutics Inc., a Swiss firm with research operations in Cambridge — agreed to form an intellectual property alliance with academic research institutions on both sides of the Atlantic that claim to be the first to discover CRISPR-Cas9. The pact allows them to share the science behind that system as they move forward with developing medicines.

Editas was not a party to that agreement, chiefly because the intellectual property it is licensing from the Broad Institute is being challenged by the companies in the new alliance — which also includes Caribou Biosciences Inc. of Berkeley, Calif., and ERS Genomics Ltd. of Dublin. All have licensed their CRISPR-Cas9 technology from the University of California Berkeley's Jennifer Doudna and Emmanuelle Charpentier at Vienna University in Austria.

A spokeswoman for Intellia declined to comment on the new Editas licenses, citing the ongoing litigation. A representative of Crispr Therapeutics didn't respond to a request for comment.

In its new set of licenses, Editas signed agreements to use the Cpf1 technology with the Broad Institute, Harvard University, the Massachusetts Institute of Technology, Wageningen University in the Netherlands, and the University of Tokyo. It signed new Cas9 licensing deals with the Broad, Harvard, MIT, and the University of Iowa.

The broader set of licensing will make Editas a more attractive partner for other drug makers wanting to collaborate on gene-editing research in specific therapeutic area, Bosley said.

Editas, founded in 2013, was the first CRISPR-Cas9 company to go public, raising $94 million in a February initial public offering. The company has more than 90 employees and recently moved into a 68,000-square-foot headquarters and research space outside Kendall Square. Bosley said the company will continue expanding in 2017.