This is Why the Decision on Who Invented CRISPR Matters

February 16, 2017
By Alex Keown, BioSpace.com Breaking News Staff

WASHINGTON – On Wednesday, the crucial question of who owned the patents for genome-editing technology CRISPR-Cas9 was decided. The U.S. Patent Office ruled the Broad Institute of Harvard University and the Massachusetts Institute of Technology were the owners.

The Broad Institute had been contesting ownership of the patents with the University of California. In the ruling, the judge allowed Broad’s original CRISPR patents to stand, The Atlantic reported. Initial work on CRISPR was done by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier.

"CRISPR" refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the "molecular scissors" that cut and edit, or correct, disease-associated DNA in a cell. Gene therapy essentially transforms cells inside a patient to harness their immune system to fight an invading disease on its own. CRISPR-Cas9 is considered revolutionary technology, and as such is likely, at some level, to be used by many companies and institutions.

The technology was first described by Doudna and Charpentier in the journal Science in 2012. However, Feng Zhang, a researcher at the MIT-Harvard Broad Institute, was the first to win a patent following his submission of laboratory notes to prove he was the first inventor. The University of California contested the patent in a 2015 lawsuit. On Wednesday, the judge ruled Zhang’s research into editing the genomes of mouse cells “would not have been obvious” based on the original work done by Doudna and Charpentier, Stat News reported. The judge’s reasoning was “because one of ordinary skill in the art would not have reasonably expected a CRISPR-Cas9 system to be successful” in those higher-order cells, Stat reported.

Now that the patent issue has been settled, the next question will be how does this ruling effect the work being done by the biotech industry, as several companies have licensed CRISPR-Cas9 products from both The Broad Institute and UC Berkeley. In the immediate, share prices of Intellia Therapeutics and Swiss-based CRISPR Therapeutics fell after the ruling. Both of those companies licensed the technology from UC Berkley, Gizmodo said.

Broad has licensed the technology to Cambridge, Mass.-based Editas Medicine for therapeutic applications and provided non-exclusive licenses for other CRISPR applications to GE Healthcare, Monsanto and Evotec, Gizmodo said.

Shares of Editas soared 29 percent after the ruling was issued, Stat News said.

Following the ruling, the Broad Institute issued a statement that it will allow academic researchers to use the CRISPR technology for free, but companies will have to pay for the right.

“We believe CRISPR should continue to be available to the global scientific community to advance our understanding of the biology and treatment of human disease, and to help lay the groundwork for a new generation of therapies,” the Broad Institute said, according to Gizmodo.

There are currently 50 patents that have been issued by the U.S. Patent office that are connected to the CRISPR technology, 14 of which are owned by the Broad Institute, Gizmodo reported.

While the Patent Office ruled, there is the chance Doudna and UC Berkley could appeal.

In November, an oncology team in China became the first to inject cells that contained edited genes using CRISPR-Cas9 technology into a human patient. The Chinese team, helmed by oncologist Lu You, removed immune cells from the lung cancer patient’s blood and disabled a gene using the CRISPR-Cas9 technology. The gene codes for the PD-1 protein, which functions as an immune checkpoint. The cells were edited and re-injected back into the patient without the PD-1 protein in hopes that the cells will attack and destroy the cancer.

The first CRISPR trial in the U.S. is slated for this year, backed by The Parker Institute for Cancer Immunotherapy. The gene editing technology will be used to alter a patient’s blood cells particularly the T cells, in order to kill the cancer cells.