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Biological Chemistry

The CRISPR craze continued

Patents were disputed, companies went public, and CRISPR-edited cells made it into humans

by Ryan Cross
December 14, 2016 | A version of this story appeared in Volume 94, Issue 49

Credit: Ian Slaymaker/Lauren Solomon, courtesy of Broad Institute
Guide RNA (green) directs the Cas9 enzyme (space-filling structure) to a specific site in DNA (red) for editing.
The guide RNA directs the Cas9 enzyme to a specific DNA location for gene editing.
Credit: Ian Slaymaker/Lauren Solomon, courtesy of Broad Institute
Guide RNA (green) directs the Cas9 enzyme (space-filling structure) to a specific site in DNA (red) for editing.

The buzz over CRISPR/Cas9 gene-editing technology didn’t diminish in 2016. The year was chock-full of scientific advances and biotech investments, culminating in a U.S. patent court hearing that pitted pioneers in the field against one another to determine who holds the licensing rights to CRISPR.

U.S. Patent & Trademark Office judges heard arguments earlier this month from attorneys representing the Broad Institute of MIT and Harvard University about why its patent—which specified the first use of CRISPR in eukaryotic cells such as human cells—should win the fight. The University of California, Berkeley, and the University of Vienna disagreed. Although their initial patent, filed but not approved before Broad’s, described using CRISPR in prokaryotes such as bacteria, their attorney argued the obvious next step was to translate the technology into eukaryotes. “There was no special sauce” in Broad’s eukaryote CRISPR patent, he stated.

Timing for the final decision is unknown, but that isn’t stopping biotech firms from pushing ahead. Editas Medicine became the first CRISPR franchise to hit the stock market in February. Intellia Therapeutics followed, forming a partnership with Regeneron Pharmaceuticals in April and going public in May. CRISPR Therapeutics went public in October.

Meanwhile in the lab, multiple research groups reported different CRISPR strategies for potentially curing sickle cell disease in human cells and animals. Scientists at Harvard also created an improved CRISPR method to precisely modify single bases in DNA.

CRISPR got into humans this year too. After receiving NIH approval, University of Pennsylvania researchers anticipated being the first to use CRISPR editing on immune cells that would be injected into cancer patients. But in October, Chinese researchers beat them to the punch, triggering a Penn scientist to say this could lead to “ 2.0.”

Looking ahead to 2017, researchers hope to hear a decision on the CRISPR patent case. They are maybe a little less hopeful about Jennifer Lopez’s possible TV show, a thriller about CRISPR-based bioterrorism attacks.


Top Headlines of 2016

Top Research of 2016

Revisiting Research of 2006


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