gene editing DNA cutting scissors wildpixel/iStock/Thinkstock

Patent awarded for DNA-targeting complex at heart of CRISPR-Cas9

This and recently granted U.S. patent cover how to make and use gene editing scissors in any setting.

The University of California announced Oct. 30 that the U.S. Patent & Trademark Office has granted U.S. patent 10,113,167, covering unique RNA guides that, when combined with the Cas9 protein, are effective at homing in on and editing genes. These RNA/protein combinations act like precision-targeted gene editing scissors.

This CRISPR-Cas9 DNA-targeting complex — discovered by Jennifer Doudna, Emmanuelle Charpentier and their teams at the University of California-Berkeley and the University of Vienna in Austria — is one of the fundamental molecular technologies behind the revolutionary CRISPR-Cas9 gene editing tool, the University of California said.

Together, this patent and prior U.S. patent 10,000,772 cover CRISPR-Cas9 compositions that are useful as gene editing scissors in any setting, including animal and human cells, the announcement said. Additionally, the claimed CRISPR-Cas9 guides work both as two distinct pieces of RNA or as a simpler system involving a single piece of RNA.

"These gene editing scissors have already sparked countless research projects across the globe to improve health and the food supply," said Edward Penhoet, special adviser to the University of California-Berkeley chancellor and special assistant to the president of the University of California. "These patents are the first of many that we expect to be awarded for the Doudna-Charpentier teams' groundbreaking invention."

The newly issued patent also encompasses protein-RNA compositions that can deliver CRISPR-Cas9 into cells in two different ways: as a fully functional ribonucleoprotein (i.e., Cas9 protein complexed with RNA), and with the components encoded by DNA that is subsequently expressed and assembled inside the cell to form a functional CRISPR-Cas9 complex, the university said.

According to the University of California, patents for the use of CRISPR-Cas9 for gene editing in all types of cells have already been issued to the Doudna-Charpentier team by the European Patent Office (representing more than 30 countries), the U.K., China, Japan, Australia, New Zealand, Mexico and other countries.

The University of California said it has a "long-standing commitment to develop and apply its patented technologies, including CRISPR-Cas9, for the betterment of humankind." Consistent with its open licensing policies, the university system allows nonprofit institutions, including academic institutions, to use the technology for educational and research purposes.

In the case of CRISPR-Cas9, the University of California has also encouraged widespread commercialization of the technology through an exclusive license with Caribou Biosciences Inc. of Berkeley, Cal., which has sublicensed the technology to many companies worldwide, including to Intellia Therapeutics Inc. for certain human therapeutic applications. Additionally, Charpentier has licensed the technology to CRISPR Therapeutics AG and ERS Genomics Ltd.

According to the announcement, the newly granted U.S. patent (10,113,167) is not involved in any interference proceeding before the Patent Trial & Appeal Board of the U.S. Patent & Trademark Office or appeal before the U.S. Court of Appeals for the Federal Circuit.

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