Gene Editing, Market

Gene editing stocks get bushwhacked as new studies highlight CRISPR/Cas9 cancer risks

Gene editing stocks have a habit of running up and down when some new piece of research underscores their potential or the risks associated with the tech.

Today the numbers were plunging into the red for CRISPR Therapeutics $CRSP, Intellia $NTLA and Editas $EDIT as some top-level investigators spotlighted a connection between CRISPR-Cas9 and an added risk of cancer associated with the technology. And soon after the studies hit, the biotechs involved began to strike back at the researchers’ conclusions.

Jussi Taipale

The problem, says Professor Jussi Taipale, now at the Department of Biochemistry, Cambridge, is that editing cells with p53 is hard because it activates a system that flags the cell for DNA damage. As a results, there’s a natural preference for cells that lack the p53 pathway, which are more vulnerable to cancer, giving rise to vulnerable cell populations. 

Depressing p53 activity in cells may well have the same risks, as they are stripped of their natural defenses.

Taipale worked with investigators at the prestigious Karolinska Institute in Sweden.

“Although we don’t yet understand the mechanisms behind the activation of p53, we believe that researchers need to be aware of the potential risks when developing new treatments,” Taipale says. “This is why we decided to publish our findings as soon as we discovered that cells edited with CRISPR-Cas9 can go on to become cancerous.”

Simultaneously, a group at the Novartis Institutes for BioMedical Research also raised the same issue with p53, underscoring some of the risks involved with CRISPR/Cas9 technology and spooking investors who were gambling that this technology will deliver a whole new generation of drugs.

Darren Griffin

Darren Griffin, a genetics expert at Kent University who was not involved in either study, told Reuters that the study raises “reason for caution, but not necessarily alarm”.

“Almost any treatment that has the power to do good, has the power to do harm and this finding should be considered in this broader context,” he added. “As we learn more about the CRISPR-Cas9 system and how it can be used, this study will inevitably be considered a significant finding.”

Intellia was one of the first to respond to the report, and the drooping stock price. In an email to me the biotech noted:

We’ve observed no signs of this type of toxicity or cells transforming into cancer or tumors in Intellia’s in vivo and ex vivo programs.

Intellia has not found this type of effect in any of our in vivo studies using our lipid nanoparticle delivery system, including our 52-week study of successful TTR knockdown in mice and our ongoing studies of non-human primates. For Intellia’s ex vivo work, we have achieved efficient editing (>90 percent) in HSCs and T-cells and have not seen the type of toxicity or tumorgenicity being reported in these papers. Despite extended observation in animals and in vitro cultures, we have not seen this effect. Intellia’s current approaches are directed at different cell types.

CRISPR shares were down 13% Monday afternoon, Intellia was off 9% and Editas shed 10% of its value.


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