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We’ve been able to take exactly that same motif, exactly that same protein structure into the laboratory and build out a library of proteins that allow us to target with singular specificity any endogenous gene. And so this is our core technology. And we can use this to drive biologies from the DNA, at the DNA level. And it’s that opportunity which really differentiates us and defines our strategy and business model going forward.So our internal focus is to employ this technology in the area of human therapeutics, but we’ve also been quite successful in leveraging this into areas outside of human therapeutics in partnerships in the area of life science research, as well as in plant agriculture. And I’ll cover those at the end of the presentation. And lastly, just from an overview perspective, we dominate the intellectual property in this area, and because of our partnerships and because of our business model, this hybrid business model, we’ve been able to grow the business quite significantly while retaining a very strong cash position and a very clean balance sheet. And I’ll come back to that near the end of the talk. But first, just one slide on the core technology. As I mentioned, we employ this class of DNA-binding proteins called zinc finger DNA-binding proteins. And again, without going through all of it, we simply have been able to take what nature created and develop this and all manage it in a way that in the laboratory we can use this to assemble zinc fingers and can target exactly the gene we want. But what we want to do is drive biologies. And so we can link these targeting proteins, these zinc finger proteins that bind to a specific sequence of DNA to what we would call functional domains. So we can use this to create zinc finger transcription factors that allow us to turn on or turn off the expression of a gene and we can link these with molecular scissors, the so-called nucleases to allow us to induce or create a double-stranded break at a very specific site in the genome and then we can drive the resolution of that double-stranded break in several ways. We can cause that gene to be put back together in a way that’s dysfunctional, that losses some of the nucleic acid. This causes a disruption in the gene expression, and this is what we’re doing in our HIV program.
We could also use this to actually change DNA sequences or correct them, and much of our monogenic disease particularly in the areas of hemophilias are involved with this. We could also and finally use this to insert large sequences of DNA into so-called safe harbor locations in a genome. We can do this in plants, we can do this in humans, and so it’s a very broad, very general technology platform that has very unique opportunities in the area of human health care.And so, when you think about the technology platform and you start to filter that through the kinds of therapeutic programs that can be developed, we can think about this in two large buckets, direct in vivo applications and modified cells or ex vivo applications. And while I won’t go through all of these today, this begins to give you a sense of how we think about the opportunity to leverage this very general, very robust, gene-specific, gene-modification, gene-regulation technology platform to create highly differentiated, and in many cases, therapeutics that are not just intended to treat diseases, but actually physically and permanently correct or change DNA sequences in a way that can engineer genetic cure. And that’s really the ultimate goal of the technology. Read the rest of this transcript for free on seekingalpha.com