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And so in organisms such as man, where we have a large number of genes compared to yeast, this is the most abundant type, the most common type, of DNA binding protein, and the most common type of protein that regulates DNA. And so what we’ve been able to do is really take advantage of this protein structure, this protein motif, and build out a library at Sangamo that allows us to prospectively build a zinc finger protein to target any DNA sequence we want with singular specificity.And then using that to target exactly that gene sequence, we can bring into the context of that gene proteins that allow us to drive biology at the DNA level. And this is a fundamentally unique approach in drug development, in agriculture, in generating unique cell lines or transgenic animals, because it’s the only platform that allows you to drive unique biologies at the DNA level. So that’s the thing that really differentiates us. What we’re doing, then, is applying us in the area of novel drugs. And instead of looking at novel treatments that might function at the protein level by blocking a receptor for a certain period of time or something like that, we’re actually looking at driving outcomes at the DNA level, which allows us to talk about curing diseases, actually changing the DNA in a way that actually cures, not just treats, diseases. And so those are the two fundamental elements at Sangamo. Because I think if you walk out with nothing else, that’s the critical view from our perspective. From a business model point of view, and I’ll go into this in a bit more detail, we’ve been very successful in leveraging this core technology in areas outside of human healthcare, and as a matter of fact, I think in about an hour from now our collaborators at Sigma-Aldrich will be presenting, and you’ll hear a lot more about zinc fingers and the work that they’re doing in the area of research reagents and transgenics.
We also have a significant collaboration with Dow Agrisciences in the ag space. And those collaborations, along with a very successful partnership that we just announced with Shire, have allowed us to operate the business in a very different way. For instance, we started this year with $85 million in cash and while prosecuting Phase II clinical trials on our own, bringing forward a pipeline of preclinical programs, we’ve guided to ending the year with at least $75 million in cash, because of this diversified business model.And our costs, both in terms of dollar outlays as well as intellectual outlays, for those revenues that come in from Sigma and Dow, is essentially zero. They’re running those businesses and we receive milestones and royalties. And finally, in all of this, we have an absolutely dominant intellectual property position. So that’s the overview. I think that’s the critical points to understand. Very quickly, this is what I just told you about in terms of the technology platform. We have the ability to engineer DNA binding proteins, zinc fingers, to target exactly the gene we want, and then we can use this to drive biology. So we can use this to regulate gene expression. And our most advanced program in activation of a gene is in the Parkinson’s field, where we’re now in non-human primate models. So in monkey models of Parkinson’s, where we’re activating the endogenous GDNF gene, the gene that has been shown have a dopaminergic effect. And by activating the endogenous gene, we don’t end up with any of the immunological problems that are seen with the recombinant protein. From a repression perspective, our most advanced effort there is again another neurological indication, in Huntington’s disease. And that’s a very active effort for us. So that’s the gene regulation side of things.
We can also use this technology to edit genes, to physically change endogenous genes. And so one of the things that’s probably the most relevant set of data that drove our new Shire collaboration was correcting a gene. And we showed this in two years of presentations at ASH, and a most recent Nature Medicine paper where with a single treatment, a single injection of a zinc finger nuclease targeting the factor 9 gene in a mouse model of hemophilia B, we were able to correct that gene, change the DNA sequence, and get the correct protein, and normalize the coagulation timeframes in this mouse model of hemophilia. And arguably that’s the principal data that drove our Shire deal.Read the rest of this transcript for free on seekingalpha.com