- Two independent studies by Novartis and Karolinska Institute showed that CRISPR-Cas9 may increase the risk for cancer by disrupting function of the essential p53 DNA repair protein.
- CRISPR Therapeutics‘ stock was down 15.3% Monday morning, while Editas and Intellia, two other companies developing CRISPR-related therapies dropped 7% and 8% respectively.
- This finding only affects some of CRISPR’s therapies.
There could be a big issue with a revolutionary gene-editing tool.
CRISPR-Cas9 is a gene-editing tool made up of a group of molecules that can find a specific gene in a cell and alter it. In theory, it’s supposed to be able to tackle genetic-based diseases like cystic fibrosis and cancer.
However, two independent articles published by Novartis and Karolinksa Institute in the journal Nature now report the application of the CRISPR-Cas9 may inadvertently increase the risk of cancer by hijacking proper functioning of the p53 protein. Mutation of the DNA-repairing p53 accounts for a large proportion of cancers.
The CEO of CRISPR Therapeutics said in a comment to STAT News that while the results are plausible, it may apply to the DNA replacement function of CRISPR more than the DNA excising function of the gene-editing tool. However, he said there will be increased scrutiny as CRISPR develops and is tested to make sure it doesn’t turn cells cancerous.
In response to the new data, CRISPR Therapeutics’s stock was down 15.3% Monday afternoon, while Editas and Intellia, two other companies developing CRISPR-related therapies dropped 7% and 8% respectively.
“We’ve observed no signs of this type of toxicity or cells transforming into cancer or tumours in Intellia’s in vivo and ex vivo programs,” Intellia said in an email statement to Business Insider.
Researchers at the Karolinska Institute suggest inhibiting p53 may help gene editing run more smoothly. In a statement to Business Insider, Editas defended its current approach.
“For making medicines, we believe inhibiting p53 to increase HDR is not appropriate or needed as it could allow the accumulation of unwanted mutations from non-CRISPR mechanisms, and we can now achieve much higher targeted integration rates and gene correction without suppressing p53 than the authors of this paper saw in their experiment.”
This isn’t the first time speculation has surrounded the gene-editing therapy. In January, concerns over immune response to infection caused the stocks to take a dip. In May, the FDA put a hold on the first clinical trial of CRISPR before it was underway.
Although this finding presents a problem for stem cells and other therapies which trigger the p53, it does not seem to affect therapies that edit T-cells to treat cancer, for example.
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