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Sangamo BioSciences, Inc. (SGMO)
30th Annual J.P. Morgan Healthcare Conference
January 12, 2012 10:30 AM EST
Edward Lanphier – President & CEO
Ipsita Smolinski – Capitol Street
Ipsita Smolinski – Capitol Street
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Thank you, very much. Thank you all for coming out early on the last day. I thought I’d start since it is the last day, maybe just a little more informally and give you a little color on our week, it’s been as always a very productive week but this year I think there’s been at least from my perspective a different level of recognition for Sangamo, the collectively the team has had nearly 100 meetings during the week with the whole lot of the ecosystem that grows up around this conference with analysts and investors and bankers and probably the most ubiquitous new constituency, the security guards are – we’ve seen a lot of them.
But we’ve also had a very productive interaction with the so many pharmaceutical companies that are camped out around the conference and I think as I said there’s been a different level of recognition for the company and its really driven by and with all the meetings we have, there’s a clear signal, there’s a clear consistency of it and its driven by two things. One, an enthusiasm for HIV program and particularly driven by the data that were presented in September at ICAC and then the manifestation or direct consequences of those data for the two new phase, two trials that we announced on Monday.
And the second is a deeper understanding, a deeper appreciation and enthusiasm for our Zinc finger nuclease platform and its unique ability to address monogenic diseases. And I will talk a little bit more about that, but the recognition of what we can drive in terms of engineering genetic cures I think has been most recently presented at ASH in terms of our factor 9 data and I will cover that. So it’s been as always a productive week, but this week in particular I think for us has been unusually positive and I look forward to telling you more about that.
So my presentation will contain forward-looking statements. I’ll refer you to our forms 10-K and 10-Q file with the SEC. Again as I think most of you know, Sangamo was focused on a unique technology platform that allows us to engineer proteins Zinc finger DNA binding proteins to target exactly the DNA sequence we want and then use that ability to target the DNA to drive unique biology’s and I’ll obviously talk more about that.
Our goal internally is to apply this to the development of novel therapeutics by functioning at the DNA level, we can absolutely drive unique genotypes and subsequent clinical phenotypes and it’s that opportunity that I think is of greatest focus for us.
We’ve also been quite successful in leveraging this science into areas outside of therapeutics, partnerships with Dow AgroSciences in the plants space and with Sigma-Aldrich in the Research and Transgenic Animal space; I’ll briefly cover those at the end. But let me emphasize because you will hear about this in our goals that our business model including in the therapeutic space is to move things forward, establish their value and then move forward with partnership. And one of our major goals this year is to establish our first major therapeutic partnership. And lastly, as a consequence of our unique business model and our ability to finance last year, we ended the year with guided ending the year with approximately $85 million in cash and ending this year, assuming no partnerships with at least $60 million in cash and so a strong balance sheet.
So very quickly just to summarize the technology platform as I mentioned we can engineer Zinc finger proteins to target precisely the DNA sequence we want and then we can use that to drive biology’s by joining those Zinc fingers with so called functional domains that allow us to turn on or turn off an endogenous gene and linking these with enzymes nucleases that allow us to specifically target the DNA sequence and change that DNA sequence. And it’s a lot of the Zinc finger nuclease technology that I am going to be talking about today.
As I mentioned this platform is agnostic to the DNA sequence, is agnostic to the species and so we’ve been very successful in leveraging this outside of the therapeutic space with partners, but we are focused in the area of Human therapeutics and our pipeline is maturing. I will spend time today really focused on our HIV program and as I mentioned earlier our monogenic disease program and in particular the Hemophilia and Hemoglobinopathies.
So the HIV program is really a pipeline in a product, we are using our Zinc finger nucleases to target CCR5; I’ll discuss that gene target in a moment. Initially in T cells but also in hematopoietic cells. And I am not going to spend any time today talking about that program, the best available information on that I think is the presentation made by Paula Cannon at CROI and that presentation is still up on the CROI website.