Synopsis:
* May have a "functional cure" for HIV, meaning that, the person will still be infected with the virus but will no longer have to take daily "AIDS cocktail" medication to stay healthy and prevent the progression to AIDS. Possibly rendering some of $GILD's and $BMY's products obsolete.
* A short-term catalyst is coming up at CROI (Conference on Retroviruses and Opportunistic Infections), where sometime between Feb 27 - Mar 2, 2011, Sangamo will present the results from two (2) Phase 1 clinical trials on their HIV therapeutic. CROI Dates. And look for the words "March" and "CROI" on the transcript of their latest conference call.
Here is a Bloomberg TV video clip from Friday Feb 11th by the lovely Shannon Pettypiece .
This article may get a little bit technical but please bare with me as this technology is so profoundly different from what any other Biotech out there has that I implore you to take the time to understand it.
Sangamo Biosciences has a technology, called zinc finger DNA-binding proteins (ZFPs), for modifying DNA in living human cells. You'll remember that, DNA is just a very long string of As, Ts, Gs, & Cs that code for the proteins that make up our bodies.
For example: “AAAA TTT GGG CCCC TTTTTTTT GG CCC A GGGGG”.
Might tell a cell to: "Code for the CCR5 receptor in CD4 T-cells".
To help you better understand what the zinc fingers are doing, indulge with me on this metaphor. Just as you can highlight a string of letters on your laptop, when you are typing, and can then press the delete button to erase what was highlighted, Sangamo can do the same thing to DNA. Imagine for a second that the genetic code is one huge string of letters on a Microsoft word document. Essentially what the ZFPs are doing is finding the exact "word" (read: DNA sequence), "with singular specificity", and then pressing the delete button. The ZFPs of course are doing this to the actual DNA of real, living, breathing human cells. To really help you understand how the ZFPs work watch this video produced by Sigma Aldrich which licenses the ZFP technology from Sangamo for their CompoZr genome editing.
The CCR5 receptor in T-cells, the white blood cells that are an integral part of the immune system & the ones that get destroyed by HIV, is what allows the HIV virus to enter the cell. Without the CCR5 receptor, on the T-cell, HIV cannot infect the T-cells.
Let us continue with our typing metaphor. If you were to highlight the words "Code for the CCR5 receptor in CD4 T-cells" in the DNA and press the delete button, you would remove the instructions that tell the cell to produce the CCR5 receptor making it immune to HIV and this is exactly what Sangamo has done to the T-cells of the HIV patients in their SB-728-T trials.
Anecdotally, we already know that it works (at least in 1 person), as quoted from a BusinessWeek article written by Rob Waters :
"One person who hopes it will prove effective is Matt Sharp, 54, an AIDS educator who was diagnosed in 1988 and today takes a daily regimen of three antivirals. He learned about the Sangamo trial a year ago and enrolled. Since last summer, when Sharp received an infusion of his own gene-modified T-cells, blood cells that help the immune system fight infection, the number of those cells has doubled, he says. "I'm just hoping I could get an infusion once a year that would keep HIV under control and I won't have to deal with the effects of taking medication.""
Additionally, SGMO issued a press statement of some preliminary Data on Jan 19, 2010 which substantiates the claims made by Mr. Sharp. From the SGMO press release: "In fact, we calculate that more ZFN-modified cells were present at 20 weeks than were initially infused... Importantly, ZFN-modified cells expanded over the period that we monitored the subject and were well tolerated." http://investor.sangamo.com/releasedetail.cfm?ReleaseID=438350
The bottom line is: I am long SGMO stock betting that they will present positive data at CROI but we must remain fully-aware that if the data is not positive the stock could absolutely get destroyed. Remember this is still Phase 1 data. You need to have a strong stomach to play around in and handle the volatility that the call options known as "biotech stocks" can dish out. Be careful out there.
Follow me on Twitter @Elwalvador
Disclaimer: In no way should anything that I say or type be taken as a recommendation to buy or sell any security. I am merely stating what I am doing with my money. What you do with your money is your responsibility. Trading stocks and options is extremely risky and you could lose all of your money.
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