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TheStreet Open House

Sangamo BioSciences' CEO Presents At The 2012 Wedbush PacGrow Lifesciences Management Access Conference (Transcript)

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.

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