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One-Size-Fits-All CRISPR Lock Could Catapult the Sector Forward

- By Matt Winkler

Gene-editing stocks may soon take a leap forward with the discovery of what is being billed as a one-size-fits-all DNA lock.

The three pure-play CRISPR stocks have been on the decline since early 2018. Shares of Intellia Therapeutics (NTLA), Editas Medicine (EDIT) and leader CRISPR Therapeutics (CRSP) have magnified the declines of the broader stock indexes late last year. This is typical for a brand new speculative industry that is still trying to find its footing and is a long way from any sort of stable revenue. Still, two out of the three, CRISPR and Editas, remain significantly higher than 2016 initial public offering levels. Editas remains up 55% and CRISPR a hefty 181% while Intellia lags, down 21% from its IPO price.

Depending on how the new technique discovered by Duke University researchers is applied to the space, the sector could start to move forward at a faster pace. Getting gene-editing technology past the FDA is especially challenging, fraught with dangers such as clinical holds that are impossible to predict and that can damage investors severely. The main issue is that CRISPR systems are attempting to edit the code of life itself, and side effects from errors could be especially dangerous and extreme if the wrong genes are edited. The FDA is acutely aware of this, which means setbacks could crop up at any moment at the first sign of trouble.

While new and improved CRISPR systems are being continually developed that have increased the accuracy of targeted gene editing, the other big problem is that adopting an entirely new enzymatic system would require fundamental retooling at each company. The costs of doing so are too high, meaning that CRISPR-focused companies are stuck with the systems they are already using.

Duke University researchers may now be able to help address this problem by developing a one-size-fits-all hairpin lock system that could increase the accuracy of any CRISPR system by at least 50-fold. As it stands now, CRISPR looks for a 20-nucleotide long complementary match that locks a guide RNA into place and induces the enzyme attached to make its cut. But, cuts can still be made if one or two nucleotide base pair is off, risking cutting out the wrong genes.

The hairpin lock works to ensure that the match has to be perfect before a cut is made in the genome. It does this by doubling the size of the guide RNA to 40, which then folds back on itself and binds to itself, preventing any accompanying editing enzyme from binding on to the DNA while the lock is in place. Then, if the match is perfect, the lock previously binded to itself instead binds to the matched DNA, unfolding and allowing the accompanying enzyme to initiate the cut. Think of it like a double lock on a door for extra security. Since the double lock does not affect that cutting enzyme itself but only the guide RNA, it could potentially be applied to any CRISPR system because all of them use guide RNAs to find their targets.

The double lock system has been tested on five different CRISPR mechanisms so far, and accuracy has been improved by an average 50-fold. If this innovation can be applied across the systems currently in use by CRISPR-focused companies, then it could significantly help in allaying fears of editing mistakes, make clinical holds less likely, and help all companies in the space move forward with their efforts.

Disclosure: No positions.

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This article first appeared on GuruFocus.