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Sangamo Biosciences, Inc. (SGMO)
Lazard Capital Markets 9th Annual Healthcare Conference Call
November 13, 2012 3:30 pm ET
Edward Lanphier – President and Chief Executive Officer
Ryan S. Martins – Lazard Capital Markets LLC
Ryan S. Martins – Lazard Capital Markets LLC
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and he is going to give a presentation for about ten minutes and we’ll just head into some Q&A. So on to you.
Thank you, Ryan and thank you for including Sangamo in this year’s conference, under having a shameless start off, I want to wish my father a happy 80th birthday today. I think that’s pretty correct handing there.
As Ryan said, I’m going to take ten minutes that thirty slides I’m going to go through some very quickly just very high level, and because more trust in me and certainly more interesting for me will be a chance to take questions and talk about the Company and talk about where we’re going.
The presentation will contain forward-looking statements, and I’ll refer you to our forms 10-K and 10-Q that filed with the SEC. So again, these are the bigger slides are up on our website, for those of you are familiar with the Company, I won’t go through it, but the real critical piece to walk away from any discussion about Sangamo is our core technology, our ability to engineer a class of naturally occurring DNA-binding proteins called the zinc finger proteins to target exactly the DNA sequence for a while. And then this is the key concept, throughout biology at the DNA level. And that is unique to Sangamo, it’s unique to really developing therapeutics. And so that’s the critical piece to know about, and I’ll come back to this at the end.
And this is just one slide on zinc fingers again, highly conserved. this is how most genes on our planet are regulated and in most species and what we do is use the natural modularity of these proteins to target exactly the sequence we want. And then wait them with an appropriate functional domain to drive that biology and I’ll try to talk about what those are as we go through that.
And so if you think about this, this is a toolbox. We can target exactly the gene we want. and then on the left-hand side of the slide, we can use it to turn on or turn off for the endogenous gene, the gene inside the patient or we can use it on the right-hand side of the slide to actually physically change the nucleic acid sequence, stop a gene from making a protein or to change the nucleic acids in a way that it makes it different protein. And that’s particularly relevant where there’s a mistake in the gene, you want to correct that gene, and therefore make the correct protein.
So this is the toolbox that we use. but the real goal here is very straightforward and very clear to use this toolbox, to use this platform toward exquisite specificity, and that really is a critical piece as you’re thinking about outcomes that are permanent and outcomes that are at the DNA level exquisitely specific under the topology at the DNA level and really alter the course of disease or if this sets engineered genetic cures. And that's really the goal here is to leapfrog treatments to really begin to approach the idea of cures.
And that’s the standard, that’s the definition of where you should be thinking about Sangamo. So this is a slide that we want to spend more time dissecting at our December 6 Analyst Briefing, when we go through our platform and well ahead of what you might expect out of us in the next two to three years.
But essentially what you should think about is, we can take the technology for gene regulation and gene modification and drive drug therapeutic development in an in-vivo setting, we can also do this in ex-vivo setting, and several of these are highlighted in terms of where we are and some of our collaborations.
The next slide actually, this is where the rubber hits the road for us. And then hopefully, Ryan will have a chance to talk about some of these, so our HIV program, and then into the monogenic disease base. So very, very quickly, and I’m happy, we’d come back to this.
The HIV program is focused on using the zinc finger nuclease technology to knock out the CCR5 gene and T-cells, CD4 cells of people with HIV with the goal of actually causing those cells to be immune from the HIV infection, but they will come out an immune response to the virus. And to cut through all of the data to-date what we’ve been able to show is that we’re successful in doing that knocking out both of the CCR5 genes, we can have a statistically significant correlation to reduction and even elimination of viral load in HIV patients. So two ongoing Phase 2 trials and very recent data presented about a month ago and ICAAC that is very encouraging from the immune system perspective, and reconstitution of the immune system but the critical issue here on the ongoing two Phase II trials, and the guidance there is preliminary data in the first half of next year, complete data in the second half, the real focus there is on viral load and reduction in viral load.