Jennifer Doudna is urging caution in the use of genetic manipulation. But she and her colleagues are also fighting for ownership of the scientific tools that make it possible.
Through Doudna’s employer, the University of California, Berkeley, the biochemist and her colleagues are forging ahead with a patent fight, bolstered with documents published Monday, over gene-editing technology known in shorthand as CRISPR/Cas9.
I wrote about the fight in December. It heated up when the U.S. Patent and Trademark Office in April 2014 awarded the first CRISPR-related patent to the Broad Institute and MIT for work led by the Broad’s Feng Zhang. The Berkeley group wants the PTO to reconsider that award, claiming that it had the invention first.
The PTO has not said whether it would re-open the case—a procedure that’s called “interference,” in patent-speak—but the Doudna camp has submitted thousands of pages of documentation to press its case. The documents were made available on the PTO website Monday.
A key element of the Berkeley group’s argument why it, and not Zhang’s group, deserve CRISPR/Cas9 ownership is a long written declaration from Dana Carroll of the University of Utah, who is considered a pioneer in the field of gene editing.
One of Carroll’s key arguments is that he and other scientists were able to use the descriptions of the Berkeley group—in a seminal paper published in Science in 2012 and then in the group’s patent application—to carry out their own CRISPR/Cas9 experiments in several kinds of cells, including eukaryotic cells—that is, from life forms more advanced than bacteria. “Not only do the applications provide this clear disclosure of the use of the compositions in eukaryotic cells, they also provide detailed descriptions of numerous steps that could be taken to apply the system to a eukaryotic cell environment,” Carroll wrote. (He also disclosed that he was paid for his work in support of the Berkeley patent.)
His argument rebuts a main point of the Broad patent: that the work of Zhang’s group made CRISPR-Cas9 editing a reality in eukaryotic cells and is therefore the invention when it comes to real-world uses, such as human therapeutics.
Well before CRISPR-Cas9, originally discovered as a bacterial defense mechanism 25 years ago, became a gene editing system, Carroll was working on earlier systems known as zinc finger nucleases. Sangamo Biosciences (NASDAQ: SMGO) of Richmond, CA, is the exclusive developer of zinc finger nucleases for human therapeutics, and the company now has a potential treatment for HIV infection in Phase 2. It’s the most advanced gene editing treatment in the world.
But proponents of CRISPR-Cas9 believe in its therapeutic potential, too. Doudna’s work has been licensed to a Berkeley, CA startup, Caribou Biosciences, which in turn has granted exclusive use of its technology in human therapeutics to Intellia Therapeutics, based in Cambridge, MA. Doudna’s co-inventor, Emmanuelle Charpentier, who has posts at institutes in Sweden and Germany, assigned her portion of the patent rights not to Caribou but to London-based Crispr Therapeutics, which recently established an R&D presence in Cambridge, MA.
The competing Boston group has thrown its weight behind Editas Medicine, also in Cambridge.
Nothing CRISPR-related has reached human clinical trials, but the use of CRISPR-Cas9 in basic research has spread across multiple organisms, plant and animal. Making sure CRISPR-Cas9 makes DNA cuts in the right places is a big challenge to overcome before it can be used in humans.
What’s more, fears that manipulation of the human germline—eggs, sperm, and embryos—is coming soon, not to treat disease but to improve people’s intelligence, looks, and other traits, have driven Doudna and several other prominent scientists—including Dana Carroll—to call for a moratorium on the practice. They published an open letter last month in Science. (Click here for my Q&A with Doudna about those fears, the moratorium, and the new research institute she runs on the UC Berkeley campus.)
If the PTO accepts the Berkeley group’s challenge to the Broad patent—no sure thing, despite the truckload of new material the group has provided—the interference hearing could last a long time as a PTO board of appeals would consider each party’s claims side by side.
Had the patent applications been filed just a little later, however, none of this would be possible. Because of the 2011 U.S. patent reform law, the system is no longer based on who invented first, and interference hearings will no longer be held. All this means that a patent fight that could determine ownership of what could be a crucial 21st century technology is one of the last being fought under anachronistic 20th century rules.
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