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Sangamo Biosciences Inc. (SGMO)
Sangamo BioSciences at Lazard Capital Markets 8th Annual Healthcare Conference Call
November 15, 2011 11:00 am ET
Edward Lanphier - President & CEO
I am Coleen (inaudible) introducing Edward Lanphier from Sangamo BioSciences and he will speak to you today. Thank you.
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I’ll talk about that in a moment. And that’s given us a differential business model, a strong balance sheet. We have about $85 million in cash at the end of the third quarter. We’ve guided to ending the year with approximately $85 million in cash which comes to about a $25 million burn which gives us several years worth of cash in the bank. And lastly we dominate the intellectual property in this space.
So one slide just very quickly to talk about the core technology and then I’ll move on to the therapeutic programs. As I said Zinc finger DNA binding proteins allow us to target exactly and specifically any DNA sequences we want. We can then link to these DNA binding proteins’ functional domains. Proteins allow us to turn on or turn off the expression of endogenous gene and that’s what we were doing for instance in our Parkinson’s program where we are activating the endogenous GDNF gene. We can also use this technology to actually physically change, physically modify an endogenous gene and this is particularly relevant in diseases where genes cause the diseases such as monogenic diseases and this is the program which we our most advanced clinical activities are in the area of HIV and I’ll talk about that in more detail.
So this is the core competency as DNA is DNA, we’ve been able to leverage this not only into human therapeutic programs, but into research collaborations with Sigma-Aldrich and into plant applications with Dow AgroSciences, but Sangamo owns a 100% of the therapeutic rights and that’s what I am going to focus on for this part of the presentation.
Our most advanced effort is in the area of HIV Aids and because we targeted the DNA level, the target is really the critical component here. And what we’re focused on initially is modifying T-cells and looking at this CCR5 gene. This is natural and HIV must use CCR5 or protein expressed on the surface of CD4 T-cells in order to infect that cell type. There is a natural mutation in people can trace our ancestry back to Eastern Europe, where that gene is dysfunctional. They are missing amino acids from that protein and that protein does not allow HIV to enter the cell and so our goal using the Zinc finger nuclease technology is to actually recapitulate both that genotype, so a dysfunctional CCR5 gene and that phenotype, a protein that does not allow HIV to infect the cells and if we are successful in doing this, we can create a compartment of the immune system that not only cannot be infected by the virus, but is capable of having an antiviral effect and thereby patients being able to go off their HIV medications and still control the virus or have a code functional cure. So that’s the objective of this program.
We have two ongoing clinical trials, both looking at this in a population of patients who are aviremic. So they are currently on hard therapy, have unmeasurable viral load and this allowed us to do several things, to look at the safety of this modified cell approach, to look at the engraftment of these cells, do they go in, do they circulate normally, do they traffic normally, and also what’s the effect on the immune system. And data from both of these or all of these areas were presented both at the conference for retroviruses and opportunistic infections in the first quarter and then just two months ago in September at [ICAC], but the real question in an anti-HIV approach is there an anti-viral effect and we are able to evaluate this in one of these clinical trials and that’s the trial at the University of Pennsylvania where these subjects come in, they are on hard therapy, they receive the modified cells that have the knocked out CCR5 gene.
After four weeks of those cells circulating, these patients will go on a treatment interruption or a drug holiday where they go off-drug for 12 weeks. And then go back on drug and we’re able to evaluate during that period of time when the virus goes up if there’s any anti-viral effect. And so data that were presented at [ICAC] are the following and so at the (inaudible) day zero you see that these patients come in with a well-controlled viral load, really an undetectable viral load.
At week four, they go off their drug and as expected all of these patients on this cohort see a significant increase in their viral load and then during their treatment interruption that typically plateaus, they go back on drug and the viral loads come down. But as you can see, there’s a couple of unusual observations here, the patient 205 in green actually return to an unmeasurable viral load during the treatment interruption which is highly unusual.