STAT’s story about CRISPR-edited cells often lacking a functional gene known to prevent cancer caused a bit of a stir. On Wall Street, some investors dumped shares of Editas Medicine, CRISPR Therapeutics, and Intellia (the three pure-CRISPR plays; other companies are pursuing other forms of genome-editing). Social media delivered the expected blowback, apparently because we dared to raise the possibility that it might possibly be necessary to test possibly CRISPR’d cells for possible cancer-causing changes. We’re doing an online chat about all this next week, but in case you can’t wait:
Cut to the chase: Is this the death knell for CRISPR? No. Not even close.
So why are we here? Because two studies in Nature Medicine, from two different teams of scientists studying two different uses of CRISPR in two kinds of human cells reached the same conclusion: One form of CRISPR is much more likely to successfully edit a cell’s genome if that cell lacks an intact p53 gene and functional p53 pathways (meaning the downstream molecular effects of the gene). Conversely, in cells with functional p53 genes and pathways, CRISPR has a very low probability of successfully editing the cells’ genomes.
The fuss over CRISPR Cas9 gene editing really is a replay of the enthusiasm over genomics in the late 1990s and eqrly 2000’s. I was investing in biotech then as now and the rationale for investing in “genomics” companies was hard to resist. It would apply to everything! It was like investing in steel and earth moving machines rather than railroads or dams picks and shovels rather than goldmines — sure to work where something more specific might fail, and on and on. Enthusiasts were true believers. Questioning the doctrine would result in accusations of scientific illiteracy and resistance to progress. But almost all of those companies failed and the few that survived consolidated essentially ito one pretty good but not so fantastic company (Illumina). Most people who invested in genomics lost tons of money. I resisted but couldn’t completely hold out . I put between 5 and 10 per cent of my portfolio into that stuff. I didn’t lose it all but I lost significantly. Editing is truly similar. One should value companies on real specific therapeutic scientific achievements. General principles always run into a wall of detail.
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