18 December 2017

New molecular scissors – everything and for nothing

A young company has created a new enzyme for DNA editing

Matthew Herper, Forbes, 12/16/2017
Translated by Natalia Tanyuk

Genome editing promises a revolution in the methods of treating diseases and prolonging life. It is all the more pleasant when companies find new tools for this work.

The young company Inscripta claims that it has discovered a new CRISPR enzyme that allows DNA editing. This is one of the most relevant trends in the field of biotechnology. The company considers it natural to distribute it for free.

"This is a real gift that we have prepared for society, because I sincerely believe that this technology is so important that it cannot be restrained or restricted," says Kevin Ness, executive director of Inscripta, formerly called Muse Bio and located in Boulder, Colorado.

CRISPR is an abbreviated name meaning short palindromic repeats, regularly arranged in groups, found in the DNA of many bacteria. Microbes use them as part of the immune system that kills viruses, but scientists use some enzymes with CRISPR to quickly cut DNA – this greatly simplifies the editing of the genetic code. Groups from the University of California at Berkeley, the Broad Institute of the Massachusetts Institute of Technology (MIT) and Harvard University took part in a decisive battle for the rights to the first CRISPR/Cas9 complex, which became widely used by biologists and biotech firms.

However, Ness says that the restrictions that the patent holders have imposed on Cas9 and the second CRISPR enzyme, Cas13b, make it difficult for many researchers to use them. There will be fewer restrictions on the new enzyme with CRISPR, Inscripta's CRISPR-MAD7. (MAD7 is an abbreviation of "Madagascar", the code brand name of the product.) Ness says he would like to involve "some people who are on the sidelines of this process."

How serious is the problem of restricting access to CRISPR enzymes? It's not clear. Inscripta believes that researchers will be charged only if they resell this enzyme without making any changes to it, include it in drugs or use it for production, but at the same time a small amount of royalties will be "much lower than industry standard conditions." But George Church, a professor at Harvard Medical School, one of the first researchers to work with CRISPR, says that Harvard, Berkeley and Broad universities have made the CRISPR/Cas9 complex free for all researchers. He also says that in similar cases, royalties should be expressed in single digits. But the discovery of Inscripta shows that the certainly noteworthy CRISPR/Cas9 complex is only one of the tools for researchers.

The reasons why Inscripta provides the CRISPR-MAD7 enzyme for wide access cannot be called exclusively altruistic. Part of their business model involves the production of some devices that they will be able to sell to scientists. "In my opinion, the long–term main activity that Inscripta is going to do is tools and reagents that will allow CRISPR to be used easily, quickly and in multiplex mode for gene editing, and even for high school students," says Brian Roberts, Venrock venture investor. Venrock has been actively working to raise funds for Inscripta in February. Another area of activity, according to Ness, will be the production of special CRISPR enzymes, taking into account customer requirements.

The president of Inscripta is John Stoelpnagel, who was the founder of the giant company Illumina, engaged in DNA sequencing. "I see now in genome editing as broad opportunities as those that have opened up as a result of genome decoding," says John. "We have to implement them, so we can take advantage of this greatest technology to expand its application, reduce cost and increase access to it."

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