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Sangamo SGMO and their zinc finger IP was added to Patent Play Stocks
ZincFinger,
I have seen your post on KBLB and I have found you very knowledgable on the topic. My sister is the director of the women's and infant center in R.I and a professor at Brown university. I will see her this weekend and wanted to discuss SGMO 's HIV /Aids project. She is in charge of over 30 newborns everyday. I was wondering SGMO's process could be geared for children. Any thoughts? Thanks in advance.
Can anyone tell me if the zinc fingers and the HIV project would work on childhood HIV/AIDS?
1:08AM Sangamo BioSci presents groundbreaking clinical data from ZFN therapeutic for HIV/AIDS (SGMO) 6.00 : Co presents data from all dosing cohorts of Sangamo's Phase 1 dose escalation study in subjects on highly active antiretroviral therapy who entered the study with CD4+ T-cells counts of <500 cells/mm3 after several years of therapy. Data from these presentations demonstrate: 1)A statistically significant relationship between suppression of HIV viral load and calculated levels of circulating CD4+ T-cells that have undergone biallelic modification of the CCR5 gene in SB-728-T-treated individuals who underwent a TI. CCR5 is the major co-receptor used by HIV to infect cells of the immune system. 2)The VL of one SB-728-T treated-subject decreased to undetectable levels during a TI. This subject was found to be heterozygous for the CCR5 delta-32 gene mutation, i.e. half that subject's CCR5 genes were naturally disrupted. 3)Confirmation and extension of previous observations of unprecedented improvements in overall CD4+ T-cell counts and CD4+ : CD8+ T-cell ratios, as well as engraftment, expansion, trafficking and persistence of the ZFN modified cells. 4) SB-728-T treatment continues to be safe and well tolerated.
SGMO>>Sangamo Bioscience’s Blood-Cell Gene Therapy Fights HIV Without Medication
By Elizabeth Lopatto - Sep 18, 2011 12:15 PM ET
http://www.bloomberg.com/news/2011-09-18/sangamo-s-blood-cell-gene-therapy-fights-hiv-without-drugs.html
Gene therapy developed by Sangamo Bioscience Inc. to mimic the blocking effect of white blood cells in people naturally immune to HIV helped six patients fight off the disease without drugs, a study showed.
In most people, the CCR5 gene acts as a receptor that shepherds HIV into the body’s cells. About 5 to 15 percent of the population, though, have one or two mutant copies of the gene that disrupts entry. The therapy by Richmond, California- based Sangamo used genetically modified white blood cells to imitate the effects of the mutant DNA, the study said.
The research, reported today at the Interscience Conference on Antimicrobial Agents and Chemotherapy in Chicago, may help patients who have HIV that’s resistant to current drugs, said Pablo Tebas, a researcher at the University of Pennsylvania in Philadelphia and a study investigator. It’s exciting because no serious adverse events were seen, he said.
The study found “these cells are going where they’re supposed to go,” Tebas said in an interview at the Chicago meeting. “At this stage it’s not a cure, and it’s a complicated treatment to expand to large segments of the population.”
Sangamo rose 1.2 percent, or 7 cents, to $6 in Nasdaq Stock Market composite trading on Sept. 16, after dropping 9.6 percent since the beginning of the year.
The most common side effect cited in the gene therapy study was a persistent smell of garlic, the researchers said. After the therapy, one patient who naturally had one copy of the mutant DNA maintained undetectable levels of HIV without drug use, the study found.
Mutant Percentages
About 10 percent of people have one normal and one mutant gene, and less than 5 percent have only mutant genes, Tebas said. The company has two trials ongoing using the modification method.
In the other five patients, the amount of HIV in their bodies first increased during a 12-week period in which they weren’t taking anti-viral drugs, and then the level dropped, according to the study. The patients were male, and ranged in age from 31 to 56. Three had been infected for about 20 years.
“We see a significant anti-viral effect,” said Samgamo Chief Executive Officer Edward Lanphier in a telephone interview. “That’s the big punchline here.”
In Sangamo’s process, doctors draw patients’ blood and remove white blood cells, also called T cells. They are sent to Sangamo and modified using naturally occurring proteins called zinc fingers that cut into patients’ DNA in the middle of the CCR5 gene. The modified cells are then returned to the patient through an infusion.
Base of Immunity
The therapy doesn’t remove the CCR5 protein from all of the patients’ cells, Tebas said. Instead, it provides a base of immunity that helps patients suppress the virus, he said.
The next step is to increase the number of modified cells in patients, he said.
Antiviral drugs, led by Atripla and Truvada, made by Gilead Sciences Inc., of Foster City, California, and Reyataz, sold by New York-based Bristol-Myers Squibb Co., generated $15.1 billion in worldwide sales last year, according to IMS Health Inc., a Norwalk, Connecticut-based industry research company.
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Sangamo gene therapy shows promise in reducing HIV
BY Reuters
— 1:16 PM ET 09/18/2011
* Viral load decreased in three of six trial patients
* Virus undetectable in one patient
* Details of development strategy expected on Wednesday
* Sangamo CEO has no plans for HIV treatment partnership
By Deena Beasley
Sept 18 (Reuters) - An early stage trial of Sangamo BioSciences Inc's (SGMO
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) HIV treatment found that the gene therapy reduced levels of the virus and even eliminated it in one patient with a naturally occurring gene mutation.
The very small Phase 1 trial tested the SB-728-T gene therapy, which is designed to disrupt the CCR5 gene used by HIV to infect cells of the immune system.
If shown to be safe and effective, the treatment could end the need for the antiretroviral drugs now used to keep the virus that causes AIDS in check by suppressing viral replication in the blood.
Trial results presented in Chicago on Sunday at the Interscience Conference on Antimicrobial Agents and Chemotherapy show a "statistically significant relationship between estimated modification of both copies of the CCR5 gene and viral load," said Dr. Carl June, trial investigator and director of translational research at the University of Pennsylvania's cancer research institute.
In a statement, June said the results suggest the need to increase the frequency of the modified cells in HIV-infected patients, which could lead to a "functional cure" for AIDS, but the means of achieving this have not been clarified.
Sangamo said earlier this year that a single infusion of the treatment improved immune system damage in nearly all of the subjects analyzed in the first trial of the therapy in humans.
The 10 patients in the trial were on antiretroviral therapy when the study began. After four weeks, six of them went on a "treatment interruption," during which they stopped taking antiviral medication for 12 weeks.
Viral load decreased in three of the six subjects, with one patient's viral load reduced to undetectable levels. That patient carried a naturally occurring mutation in one copy of his CCR5 gene.
Humans contain two copies of each gene, one from the father and one from the mother, which sometimes are referred to as the alleles of a gene.
"Since one copy of his gene was already disrupted naturally, twice as many of his cells were 'biallelically' modified," Sangamo Chief Executive Officer Edward Lanphier said in a telephone interview, meaning that both members of the CCR5 gene pair were knocked out.
He estimated that between 5 percent and 10 percent of HIV patients carry the genetic mutation.
Around 33 million people worldwide have the human immunodeficiency virus (HIV) that causes AIDS. Major producers of current HIV drugs include Gilead Sciences (GILD
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) and GlaxoSmithKline (GSK
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) .
Lanphier said Sangamo will move ahead with a strategy to maximize the number of cells that can be "biallelically" modified by SB-728-T. Options include targeting only the small segment of patients with mutated CCR5 genes or using "strategies that boost the amount of engraftment of modified cells."
The CEO said he plans to unveil details of Sangamo's plans at an investment banking conference in New York on Wednesday.
Lanphier emphasized that the company intends to take the HIV treatment into mid-stage trials without a partner.
"We have more than sufficient cash to push forward into Phase 2 trials," he said, reaffirming that Sangamo expects to have cash holdings of at least $85 million at year-end. (Reporting by Deena Beasley in Los Angeles)
Sangamo BioSciences Announces Presentation of Groundbreaking Clinical Data From ZFN Therapeutic for HIV/AIDS at ICAAC 2011
Data Represents "Significant Progress Toward a 'Functional Cure' for HIV/AIDS"
Statistically Significant Correlation between SB-728-T and Viral Load Reduction
Press Release Source: Sangamo BioSciences, Inc. On Sunday September 18, 2011, 12:15 pm
RICHMOND, Calif., Sept. 18, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq:SGMO - News) announced that data from its Phase 1 clinical programs to develop SB-728-T, a novel therapeutic approach for the treatment of HIV/AIDS, were discussed in two presentations at the 51st Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC). The meeting is being held in Chicago from September 17-20, 2011.
"The data obtained in our treatment interruption studies are very exciting and represent significant progress toward a 'functional cure' for HIV/AIDS," said Carl June, M.D., Director of Translational Research at the Abramson Family Cancer Research Institute at the University of Pennsylvania School of Medicine, who with Pablo Tebas, M.D., is an investigator in the Phase 1 studies of SB-728-T at that institution. "The statistically significant relationship between estimated modification of both copies of the CCR5 gene and viral load during the treatment interruption suggests that the next step is to increase the frequency of the modified cells in HIV-infected patients with the ultimate hope that if we do, we will achieve a 'functional cure' and eliminate the need for continued HAART."
In an oral presentation made on Saturday, September 17th at ICAAC, data were presented from all dosing cohorts of Sangamo's Phase 1 dose escalation study (SB-728-902) in subjects on highly active antiretroviral therapy (HAART) who entered the study with CD4+ T-cells counts of <500 cells/mm3 after several years of therapy ("immunologic non-responders"). A presentation made today described data from the second cohort of a Phase 1 clinical study of the same drug, conducted at the University of Pennsylvania and Albert Einstein College of Medicine, in subjects who entered the study with CD4+ T-cell counts of >450 cells/mm3 and underwent a treatment interruption (TI) of HAART following treatment with SB-728-T.
Data from these presentations demonstrate:
A statistically significant relationship (p<0.05) between suppression of HIV viral load (VL) and calculated levels of circulating CD4+ T-cells that have undergone biallelic modification (i.e. modification of both copies) of the CCR5 gene in SB-728-T-treated individuals who underwent a TI. CCR5 is the major co-receptor used by HIV to infect cells of the immune system.
The VL of one SB-728-T treated-subject decreased to undetectable levels during a TI. This subject was found to be heterozygous for the CCR5 delta-32 gene mutation, i.e. half that subject's CCR5 genes were naturally disrupted.
Confirmation and extension of previous observations of unprecedented improvements in overall CD4+ T-cell counts and CD4+ : CD8+ T-cell ratios, as well as engraftment, expansion, trafficking and persistence of the ZFN modified cells.
SB-728-T treatment continues to be safe and well tolerated.
"We are very encouraged by both this early demonstration of an antiviral effect of SB-728-T as well as the marked improvement in the overall CD4+ T-cell counts in treated subjects in the SB-728-902 trial," said Ronald Mitsuyasu, M.D., Professor of Medicine, David Geffen School of Medicine at UCLA and a principal investigator of Sangamo's SB-728-902 study. "While their viral loads are well controlled on HAART, these subjects experienced incomplete restoration of their T-cell counts. Improvement and preservation of the immune system is of paramount importance in HIV and those seen in this study show an improvement over that seen after several years of HAART."
"There is no other drug that has been shown to have the same dramatic effect on the immune system in this setting," stated Dale Ando, M.D., Sangamo's vice president of therapeutic development and chief medical officer. "SB-728 treatment results in unprecedented improvement in immune system health as measured by increased CD4+ T-cell levels and improved CD4+: CD8+ T-cell ratios, even in subjects that entered the trial with poor CD4+ counts. Moreover, the interesting kinetics in viral load that we observed in the first few subjects that underwent a treatment interruption has been borne out and has allowed us to correlate a statistically significant effect of the rate of biallelic ZFN-modification of the CCR5 gene on viral load during treatment interruption."
"Data presented this weekend at ICAAC suggest that, with sufficiently high levels of circulating biallelically modified cells and good engraftment, SB-728-T can provide a 'functional cure' for HIV," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "We continue to collect valuable data about the parameters essential for optimization of this novel drug candidate. Based on these data, Sangamo plans to continue to expand our clinical trials and carry out confirmatory studies in subjects who are natural heterozygotes for the CCR5 delta-32 mutation. We will also explore other mechanisms to enhance engraftment and maximize the impact of the HIV resistant cells on viral load and the overall immune system of HIV patients. The ground-breaking clinical data that we and our collaborators are generating continue to validate our highly specific ZFN technology as a platform for development of novel therapeutic products."
Clinical Trial Data Summary
Abst.# H2-794a: "HAART Treatment Interruption following Adoptive Transfer of Zinc Finger Nuclease (ZFN) Modified Autologous CD4+ T-cells (SB-728-T) to HIV-infected Subjects Demonstrates Durable Engraftment and Suppression of Viral Load" Presenters: Dale Ando, M.D., Vice President, Therapeutic Development and CMO and Geoffrey Nichol, M.B., Ch.B., Executive Vice President, Research and Development, Sangamo BioSciences, Inc.
In a late-breaker session on Sunday, September 18, 2011 data were presented from the six subjects enrolled in cohort 2 of the Phase 1 clinical trial of SB-728-T conducted at the University Pennsylvania and Albert Einstein College of Medicine. These subjects entered the clinical trial with CD4+ T-cell counts of >450 cells/mm3 and underwent a twelve week HAART TI four weeks after treatment with a single dose of SB-728-T.
The data demonstrate that:
A statistically significant relationship between VL and the calculated percentage of circulating CD4+ T-cells that have undergone ZFN-mediated modification of both copies (biallelic) of the CCR5 gene within a cell (P= 0.0001 for VL at the end of TI, and 0.037 for area under the curve of VL over the TI period).
A 0.8 to >2.0-log reduction in VL from peak during TI was observed in the 3 of 6 subjects with the highest estimated circulating levels of cells with biallelic modification.
One subject's VL decreased to undetectable levels such that the subject was considered to be aviremic at the end of the TI period. This subject entered the study carrying the natural CCR5 delta-32 mutation on one copy of his CCR5 gene resulting in an estimated percentage of biallelically-disrupted CCR5 genes that was twice that of subjects entering the study with wild type CCR5 genes.
Persistent engraftment of ZFN-modified T-cells, overall improvement in total CD4+ T-cell levels and CD4+:CD8+ T-cell ratios as well as normal trafficking of the cells and the safety and tolerability of the treatment.
These data provide important new information of the design of future clinical studies including enhanced engraftment strategies.
Abst.# H1-375: "Adoptive Transfer of Zinc Finger Nuclease (ZFN) Modified Autologous CD4+ T-Cells to Aviremic HIV-Infected Subjects with Suboptimal CD4+ Counts" Presenter: R. Mitsuyasu, M.D., Professor of Medicine, David Geffen School of Medicine, UCLA.
In a presentation on Saturday, September 17, 2011, Dr. Mitsuyasu discussed data from the full complement of subjects in Sangamo's ongoing Phase 1 dose-escalation study (SB-728-902) in aviremic subjects with CD4+ T-cell counts <500 cells/mm3. These subjects are often classified as immunologic non-responders because, while they have their virus levels well-controlled by HAART, their immune systems have not responded well and levels of CD4+ cells remain depressed.
The data demonstrate that:
SB-728-T is safe and well tolerated in this patient population with only mild, reversible symptoms typical of infusion reactions.
ZFN CCR5-modified cells engrafted, expanded, and persisted for the duration of the study to date (Median of 337 days, range: 115-561).
The modified cells trafficked to the gut mucosa, an important reservoir of active HIV infection.
Notably, an increase in total CD4+ T-cells was observed in all subjects and a normalization of the ratio of CD4+:CD8+ T-cells, a measure of immunologic health, was observed in the majority of subjects that entered the study with a ratio below one. These immunologic improvements were persistent over the study period.
These data confirmed and extended preliminary data that were previously reported from the first two dosing cohorts of the trial at the Conference on Retroviral and Opportunistic Infections (CROI) in March 2011.
In addition, data were presented from one subject in this trial who has undergone an extended treatment interruption one year after SB-728-T infusion and saw a VL reduction from peak during TI of >1-log, suggesting a potential antiviral effect of the treatment.
About SB-728-T
Sangamo's drug, SB-728-T, is generated by ZFN-mediated modification of the gene encoding the CCR5 receptor in a patient's own T-cells. ZFN modification disrupts the expression of this key co-receptor for HIV entry and renders cells resistant to HIV infection. The approach is based on the observation that a naturally occurring mutation in the CCR5 gene, CCR5 delta-32, provides protection from HIV infection. Individuals in whom both copies of the CCR5 gene carry the delta-32 mutation are generally not susceptible to the most common strain of HIV; those with only one copy of the delta-32 mutation are identified as "Elite Controllers" for their immune systems' ability to resist progression of HIV without the need for HAART.
Dr. Mitsuyasu and Dr. June have no financial relationship with Sangamo BioSciences, Inc.
The Next Big Thing in Biotech: Sangamo
Adam Feuerstein, senior columnist at TheStreet, explains why Sangamo Biosciences will be the stock to watch in the coming week.Sun 09/18/11 10:00 AM EST -- Gregg Greenberg & Adam Feuerstein
http://www.thestreet.com/video/11251256/the-next-big-thing-in-biotech-sangamo.html?cm_ven=RSSFeed&s=1#1166099750001
SGMO<sa explanatory article>Sangamo's Late Breaking, Game Changing News At ICAAC
15 comments | September 1, 2011 | about: SGMO
On September 17-20 the 51st Interscience Conference On Antimicrobial Agents and Chemotherapy meeting will take place in Chicago. Conference attendees have always referred to this meeting by its abbreviation; ICAAC, pronounced “ick-ack." It’s considered to be the premier meeting on infectious diseases and antimicrobial agents and is organized by the American Society for Microbiology.
A brief background that provides some context for Sangamo’s (SGMO) exciting, game changing, human clinical trial presentations at ICAAC:
CONTINUE for Full
http://seekingalpha.com/article/291210-sangamo-s-late-breaking-game-changing-news-at-icaac
Biotech Stock Mailbag: Sangamo Biosciences
BOSTON (TheStreet) -- I recently highlighted Sangamo Biosciences(SGMO_) as one of the top 10 biotech stock trades in the second half of 2010 because I perceived the pending results from a phase IIb study of SB-509 in diabetic neuropathy as a referendum on the company's zinc-finger drug technology platform.
More from Adam Feuerstein
Some readers took issue with that characterization, arguing that Sangamo and its zinc-finger technology -- which engineers custom proteins to turn disease-related genes on or off -- will not live or die based on the results from a single phase IIb study in diabetic neuropathy.
"Lest you think I'm a rabid cheerleader, let me say first that SB-509 will almost certainly fail," writes Eric B. "Your lack of insight is startling," he adds. "Zinc fingers are not drugs. The drug in the neuropathy trial are localized cells that have had their veg-f genes upregulated. The trial will determine if that is effective, not whether turning on and off genes via a zinc finger is a viable way of making drugs."
I didn't think my insight was so off base, but I do understand Eric's point. Sangamo is also developing zinc-finger "drugs" as potential therapies for hemophilia and HIV/AIDS -- the latter, especially, has garnered a lot of attention. So, Eric's right, Sangamo won't disappear if SB-509 fails to promote significant nerve growth in patients with diabetic neuropathy.
Other investors I've spoken to this week agree that the expectations around SB-509 are relatively low, so Sangamo's stock price may not take the 50% or higher haircut you'd typically see if the trial comes back negative. The more interesting Sangamo pipeline "drug" is SB-728 in HIV/AIDS, although it's also earlier stage and the clinical path forward is not entirely clear at this point.
By contrast, if the SB-509 phase IIb study is successful, Sangamo could advance into pivotal phase III trials relatively easily and also ignite the interests of potential partners. For that reason, I still view SB-509 and the upcoming phase IIb study as a very important event for Sangamo, while acknowledging the point made by Eric B. and others.
As was mentioned above, SB-509 is a protein custom engineered to turn on (or restore activity) in the gene for vascular endothelial growth factor. The so-called VEGF-A protein is responsible for nerve and blood vessel growth. [If this sounds familiar, it's because Roche/Genentech's blockbuster cancer drug Avastin blocks the expression of VEGF as a way to prevent tumors from growing new blood vessels.]
A high level of blood sugar that damages blood vessels feeding nerves is the root cause of diabetic neuropathy. Eventually, the damaged nerves lose function and even die, leading to tingling, numbness and pain. The only drugs approved for diabetic neuropathy today are painkillers and anti-depressants, none of which address the root problem of nerve damage or offer a way to reverse it.
http://www.thestreet.com/_yahoo/story/11194162/1/biotech-stock-mailbag-sangamo-biosciences.html?cm_ven=YAHOO&cm_cat=FREE&cm_ite=NA
Avg Vol (3 month)3: 492,223
Avg Vol (10 day)3: 445,489
Shares Outstanding5: 52.32M
Float: 49.64M
% Held by Insiders1: 5.75%
% Held by Institutions1: 36.00%
Shares Short (as of Jun 15, 2011)3: 8.64M
Short Ratio (as of Jun 15, 2011)3: 18.50
Short % of Float (as of Jun 15, 2011)3: 20.10%
Shares Short (prior month)3: 8.57M
setting up for short covering rally
7:17AM On The Wires (WIRES) :
Sangamo BioSciences (SGMO) announced the publication of a preclinical study demonstrating permanent functional correction of the gene that causes hemophilia B by the systemic delivery of zinc finger nucleases. The study, published in Nature, represents a significant advance in the development and systemic delivery of ZFP Therapeutics and proof of concept for ZFN-based gene-editing for the treatment of hemophilia and other monogenic diseases.
SGMO is getting close to some 6 month lows... Picked up some shares today. Hope I'm not trying to catch a falling knife!
Thanks Zinc. Very informative.
Wow Zinc, I see you're a prolific poster on the other board. What are your impressions of SGMO?
Investors Hub SGMO really got it wrong today!:
Read carefully there is nothing in the KBLB news release to indicate that the worms crossed with the larger commercial worms incorporated the zinc finger modifications.
That would be extremely unlikely IMHO because there hasn't been nearly enough time to both incorporate the zinc finger modifications AND THEN do the cross breeding.
In addition the name given to the hybrid worms, "Monster hybrid" indicates that the Monster Silk worms (i.e.: without the Zinc finger modifications) were used to make the cross.
So this is all about the commericial silk market not the high tech silk market as apparently the I Hub KBLB board well realizes. But the SGMO I V board apparently misunderstood and thought that the news was about worms incorporating the ZF modifications. And SGMO is up far more (high of 8% up so far) than KBLB (high of about 3 1/2% so far).
SGMO will probably drop back down a bit when their investors realize that this one wasn't about zinc fingers. So if anyone is about to buy SGMO I would suggest waiting a day or two to see if the price drops back when the realization that zinc fingers were not involved in today's KBLB release hits.
(Disclosure: I am about to enlarge my SGMO position myself and will certainly be waiting for such a pullback). If not I'll buy more anyway (I'm not trading just enlarging a position.)
Enjoy a bit of a free ride on KBLB, SGMO! Consider it a "tip" for the zinc fingers!
I am now inclined to expect that the KBLB CC will be strongly centered around the Monster silk developments and any discussion of zinc finger work will be done in later CCs (how much later (or sooner) is anyones guess. Could be in a few weeks, maybe a bit longer. When it does come it should be a huge boost for both KBLB and SGMO as it will be the first zinc finger product to be ready to take to market (no clinical trials required, no regulatory approval for GMO release required (and commercial silkworms (having been domesticated for thousands of years) cannot survive in the wild anyway)), no scale up of production process required (just a few short months to breed the worms to production levels) and plenty of manufacturing facilities already available with no modification needed. So a ZF product could be on the market in as little as a year from now.
Today was not the ZF PR. But soon, very soon.
disclosure: long both SGMO and KBLB
All just IMHO
Kraig Biocraft Laboratories Inc. (KBLB) Stoc
I don't own any shares but I've been watching it as well. Losses doubled for the most recent quarter compared to the same period last year and income was down as well. However, they still have $49 million in the bank and the science is very encouraging; particularly their work on HIV which is attempting to duplicate the only documented cure of the disease. In the absence of any positive news, I would expect further weakness. I will be watching closely for an entry point lower when the price stabilizes. Good luck.
Considering most people on this board are likely SGMO investors, I would appreciate anyone's opinion about the 'current' trend of the stock price.
I have done relatively little DD on the price, but I do like the technology of the company.
One is always skeptical as to an appropriate 'target price' for purchase and I'm curious about the EOY price leap and subsequent pullback.
Any thoughts would be appreciated and no I have NOT gone back a read all of the posts here, but only some.
695, i have been to your blog, it looks great! i was there around a week ago. got to much on my plate to get involved now, maybe later. i will put the sites on my favorites. thanks.
Cant get on to the SGMO CC..... had the same prob with the SIAL CC this morning. Anything exciting happen?
Check out my new Kraig Labs blog!
http://kraiglabs.blogspot.com/
I recommend you start at the top Nav bar under "more information." It links to all my KBLB DD.
Its run through the HUB of my blog network:
http://yearofthebull.blogspot.com/
Tons of rich DD on individual OTCs, Valuations & finance lessons.
i find this interesting that SGMOs deal with SIAL is tied in with KBLB. http://investorshub.advfn.com/boards/read_msg.aspx?message_id=62121220
cutting edge textiles, spidersilk made from silkworms.
Sangamo HIV project could rewrite story of state's stem cell agency
Read more: Sangamo HIV project could rewrite story of state's stem cell agency | San Francisco Business Times
San Francisco Business Times - by Ron Leuty
Date: Thursday, March 10, 2011, 2:21pm PST
A consortium that includes Sangamo BioSciences Inc. is pursuing a next-generation stem cell treatment that could provide a longer-term — if not lifetime — barrier to the AIDS virus.
But the fate of California's $3 billion stem cell research funding agency also may rest on the success or failure of the Sangamo collaboration and 13 other "disease teams" that are trying to get into clinical trials within four years.
Backed by a grant from the California Institute for Regenerative Medicine, researchers at Richmond-based Sangamo (NASDAQ: SGMO), the University of Southern California and the City of Hope medical center near Los Angeles believe they can draw stem cells out of bone marrow and knock out a gene that produces the door knob that HIV uses to access a cell. Researchers then reassemble the stem cell and place it back in the body so it can produce blood cells sans the key gene and, therefore, are resistant to HIV.
It is experimental — this specific gene therapy has not yet been tested in humans — but it has been successful in mouse models, said Paula Cannon, an associate professor of molecular microbiology and immunology at USC, who last week presented data from her lab’s work at the 18th Conference on Retroviruses and Opportunistic Infections in Boston.
The collaboration could be trotted out as a success story for CIRM, the stem cell research funding agency set up when California voters in 2004 approved of the state sale of $3 billion in bonds to finance the agency. CIRM earmarked $14.5 million in fall 2009 for the Sangamo-USC-City of Hope project.
The project received the highest science score among applicants for so-called disease team grants. Those 14 grants, totaling $230 million, were aimed at groups of researchers — academic collaborators or public-private partnerships — with stem cell-based treatments for a wide range of diseases.
The caveat? The projects must be in or on the cusp of clinical trials within four years of receiving the money.
“The clock’s ticking,” Cannon said. “There’s pressure, but there’s also pressure from ourselves to do it right and do it safely.”
CIRM, based in San Francisco, will need poster children like this to convince Californians that it has used its $3 billion wisely and is worthy of more cash. That’s a story that has been difficult to tell as CIRM’s internal follies — including the aborted selection of a new chairman at the end of last year — has seemed to grab more headlines.
CIRM has funded a research building boom — new facilities have been built at the University of California, San Francisco, and Stanford University, and are under construction at UC Berkeley. New research buildings are one thing, however, treating or curing a friend or loved one’s disease is another.
To that end, CIRM in January hired Dr. Ellen Feigal, who was executive medical director of global development at Amgen Inc. (NASDAQ: AMGN), as vice president for research and development.
“We’ve kind of been managing (the disease team projects) intensively because we want them to make their timelines,” said CIRM President Alan Trounson. “Now with Ellen Feigal, we can keep them on track and get in the clinic in the most efficient way possible.”
There’s one disease-team project that is taking cells from heart biopsies, growing them outside the body and transplanting them back; another is working on carrying cytotoxic drugs to inoperable brain tumors.
“All of them are on schedule at the moment, including some pretty interesting work in cancer,” Trounson said.
CIRM wasn’t the first money into Cannon’s project, which is about five years old. The agency initially received $100,000 over two years from the California HIV/AIDS Research Program, overseen by the University of California Office of the President, followed by money from the National Institutes of Health. Cannon’s lab started receiving the CIRM money in spring 2010.
But moving the project forward, Cannon said, would have been difficult because the generally conservative NIH likely would have deemed it too risky.
“It’s what we need,” Cannon said of the CIRM money. “It will fund us for four years so we can go to the government, the FDA, with all the preclinical information.
“Normally, Big Pharma companies are the only ones that have the money to do that regulatory work that’s needed, but this consortium is taking on the job ourselves.”
Sangamo has another gene-therapy approach in a Phase I/II trial — with investigators at the University of California, San Francisco, and UCLA — that blocks the CCR5 gene in white blood cells known as T cells.
Sangamo CEO Edward Lanphier has called it a “functional cure” for HIV.
Both SB-728, Sangamo’s T cell treatment, and the experiments in Cannon’s lab use the company’s “zinc finger” gene-editing technology. But Cannon’s work is further down the chain, changing the hematopoietic stem cells whose progeny include all blood cells, including T cells — so it could provide a more basic defense to HIV.
“Most other gene therapy strategies for HIV are all about putting something into the cell that will stop the virus,” Cannon said. “What I liked about this is, it’s just taking off the door knob (used by HIV) and you don’t have to keep taking off the door knob. Once it’s gone, it’s gone.”
Read more: Sangamo HIV project could rewrite story of state's stem cell agency | San Francisco Business Times
http://www.bizjournals.com/sanfrancisco/blog/2011/03/sangamo-hiv-aids-stem-cells-cirm.html
I've held, and been playing this one since the $4 turn, it's posted all over the Lotto board...
http://investorshub.advfn.com/boards/board.aspx?board_id=7793
Sitting with free shares now with plans to hold due to possible potential. Have played the options a few times, but they are not easy as they are not liquid enough with this low O/S
However...There has been some recent sales of shares by officers look at the filings...
7:06AM Sangamo BioSci announces four data presentations of Novel ZFN therapeutic approaches to the treatment of HIV/AIDS (SGMO) 7.99 : Co presents datat which include clinical data from two Phase One trials of SB-728-T confirming mechanistic proof of concept and expansion of ZFN-CCR5-Modified cells in presence of actively replicating virus. The data demonstrate that SB-728-T is well-tolerated with only mild, reversible symptoms typical of infusion reactions. ZFN-CCR5-modified cells exhibited durable engraftment and persistence in the peripheral blood for over a year and behaved like normal, unmodified cells in their ability to traffic to the gut mucosa, an important reservoir of active HIV infection. Demonstration of up to a 45-fold selective expansion of ZFN-CCR5-modified T-cells in this tissue, which represents levels not previously observed in adoptive T-cell approaches, also suggests that the modified cells were resistant to HIV and establish the mechanistic proof of concept for this therapeutic approach. SB-728-T treatment was also demonstrated to improve the overall CD4 T-cell count in all subjects and the CD4:CD8 ratio, a measure of immunologic health, in multiple subjects. In addition, two subjects on a brief 12 week treatment interruption after SB-728-T treatment showed interesting patterns of HIV RNA appearance and decrease prior to re-institution of HAART. Finally, consistent manufacturing at clinical scale from different manufacturing centers demonstrate that the ZFN-CCR5-modified T-cell process is robust and reproducible.
That's what piqued my interest, yes.
Gene Therapy Raises Hope For A Future AIDS Cure
MARILYNN MARCHIONE, AP Medical Writer
Posted: 11:13 am PST February 28, 2011
Updated: 11:36 am PST February 28, 2011
In a bold new approach ultimately aimed at trying to cure AIDS, scientists used genetic engineering in six patients to develop blood cells that are resistant to HIV, the virus that causes the disease.
It's far too early to know if this scientific first will prove to be a cure, or even a new treatment. The research was only meant to show that, so far, it seems feasible and safe.
The concept was based on the astonishing case of an AIDS patient who seems to be cured after getting blood cells from a donor with natural immunity to HIV nearly four years ago in Berlin. Researchers are seeking a more practical way to achieve similar immunity using patients' own blood cells.
The results announced Monday at a conference in Boston left experts cautiously excited.
"For the first time, people are beginning to think about a cure" as a real possibility, said Dr. John Zaia, head of the government panel that oversees gene therapy experiments. Even if the new approach doesn't get rid of HIV completely, it may repair patients' immune systems enough that they can control the virus and not need AIDS medicines - "what is called a functional cure," he said.
Carl Dieffenbach, AIDS chief at the National Institute of Allergy and Infectious Diseases, agreed.
"We're hopeful that this is sufficient to give the level of immune reconstitution similar to what was seen with the patient from Germany," he said.
This is the first time researchers have permanently deleted a human gene and infused the altered cells back into patients. Other gene therapy attempts tried to add a gene or muffle the activity of one, and have not worked against HIV.
The virus can damage the immune system for years before people develop symptoms and are said to have AIDS - acquired immune deficiency syndrome. The virus targets special immune system soldiers called T-cells. It usually enters these cells through a protein receptor, or "docking station," called CCR5.
Some people (about 1 percent of whites; fewer of minorities) lack both copies of the CCR5 gene and are naturally resistant to HIV. One such person donated blood stem cells in 2007 to an American man living in Berlin who had leukemia and HIV.
The cell transplant appears to have cured both problems, but finding such donors for everyone with HIV is impossible, and transplants are medically risky.
So scientists wondered: Could a patient's own cells be used to knock out the CCR5 gene and create resistance to HIV?
A California biotechnology company, Sangamo (SANG-uh-moh) BioSciences Inc., makes a treatment that can cut DNA at precise locations and permanently "edit out" a gene.
Dr. Jacob Lalezari, director of Quest Clinical Research of San Francisco, led the first test of this with the company and colleagues at the University of California in San Francisco and Los Angeles.
He warned that it would be "way overstated" to suggest that the results so far are a possible cure.
"It's an overreach of the data. There are a lot of people out there with hopes and dreams around the C-word," so caution is needed.
In the study, six men with HIV had their blood filtered to remove a small percentage of their T-cells. The gene-snipping compound was added in the lab, and about one-fourth of the cells were successfully modified. The cells were mixed with growth factors to make them multiply and then infused back into the patients.
Three men received about 2.5 billion modified cells. Three others received about 5 billion.
Three months later, five men had three times the number of modified cells expected. As much as 6 percent of their total T-cells appear to be the new type - resistant to HIV, Lalezari said.
The sixth man also had modified cells, but fewer than expected. In all six patients, the anti-HIV cells were thriving nearly a year after infusion, even in tissues that can hide HIV when it can't be detected in blood.
"The cells are engrafting - they're staying in the bloodstream, they're expanding over time," said Lalezari, who has no personal financial ties to Sangamo, the study's sponsor.
The only side effect was two days of flulike symptoms. It will take longer to determine safety, but several AIDS experts said they were encouraged so far.
"It is a huge step" and a first for the field of genetics, said John Rossi, a researcher at City of Hope in Duarte, Calif., where he and Zaia plan another study to test Sangamo's approach. "The idea is if you take away cells the virus can infect, you can cure the disease."
On Wednesday, Dr. Carl June, a gene therapy expert at the University of Pennsylvania, will report partial results from a second, federally funded study of 10 people testing Sangamo's product. He treated his first patient with it in July 2009.
Many questions remain:
- People born without the CCR5 gene are generally healthy, but will deleting it have unforeseen consequences?
- Will HIV find another way into cells? Certain types of the virus can use a second protein receptor, though this is less common and usually when AIDS is advanced. Sangamo is testing a similar approach aimed at that protein, too.
- How long will the modified cells last? Will more be needed every few years?
- Could doctors just infuse Sangamo's product rather than removing cells and modifying them in the lab?
- What might this cost?
Sangamo spokeswoman Liz Wolffe said it's too early in testing to guess, but it would be "a premier-priced" therapy - in the neighborhood of Dendreon Corp.'s new prostate cancer immune therapy, Provenge - $93,000.
Yet AIDS drugs can cost $25,000 a year, so this could still be cost-effective, especially if it's a cure.
Jay Johnson, 50, who works for Action AIDS, an advocacy and service organization in Philadelphia, had the treatment there in September.
"My results are excellent," he said. "The overall goal is to not have to take medication, and then hopefully lead maybe to a cure."
Matt Sharp, 54, of suburban San Francisco, also had the treatment in September.
"I would trade anything to not have to take a handful of medications every day for the rest of my life and suffer all the consequences and side effects," he said.
"I may not live long enough to see the cure, but I always hoped for a chance."
http://www.kirotv.com/health/27026631/detail.html
You too mkendra. I presume you heard about the HIV results?
Looks like it has potential. Must DD, first...
No, I heard about it on the radio, actually. I'll be watching...GLTY
So you saw the article over on the other board? I wonder why this company receives little attention here. Perhaps iHubber don't like spending $8 on a stock? It is up about 100% over the last 12 months. Not bad at all.
Got your PM but am on a free account. Yes, I'm new to the board. Very impressive science on the zinc finger proteins.
Amazing, this company may have a functional cure for HIV in its grasp and no one seems to be interested here. Perhaps this company is too non-volitile and well-managed for the iHub crowd.
Sangamo Biosciences Inc. Q2 2010 Earnings Call Transcript
http://seekingalpha.com/article/217154-sangamo-biosciences-inc-q2-2010-earnings-call-transcript?source=yahoo
7:07AM Sangamo BioSci announces nature biotechnology study demonstrating the use of Zinc finger nucleases to generate HIV resistant human stem cells (SGMO) 3.64 : Co announces the publication of data demonstrating the preclinical efficacy of a human stem cell therapy for human immunodeficiency virus based on its proprietary zinc finger DNA-binding protein nuclease technology. The ZFN approach enables the permanent disruption of the CCR5 gene, which encodes an important receptor for HIV infection, in all the cell types comprising the immune system that develop from hematopoietic stem cells (HSCs), and is the basis for a promising therapeutic strategy for the treatment of HIV/AIDS. Sangamo has two ongoing Phase 1 clinical trials to evaluate the safety and clinical efficacy of this approach in CD4+ T-cells.
7:01AM Sangamo BioSci presents positive Phase 2 ZFP therapeutic data from SB-509 programs in diabetic neuropathy (SGMO) 3.99 : Co announced positive Phase 2 clinical data from its ZFP Therapeutic program to develop SB-509 as a treatment for diabetic neuropathy. Data from Sangamo's SB-509-601 and SB-509-701B Phase 2 clinical trials demonstrated that SB-509 treatment resulted in clinically beneficial improvements in subjects with moderate and severe DN as compared to placebo. The data, which were described in three oral presentations, provided direct histological evidence of SB-509's dual effect on both blood vessel and nerve growth and validated the strategy of the use of multi-endpoint disease severity criteria that have been established for enrollment into the co's ongoing Phase 2b trial in subjects with moderately severe DN.
Sangamo BioSciences and Collaborators Present Data in Fifteen Presentations Highlighting Broad Therapeutic Applications of ZFP Technology
ZFP Therapeutic Programs Include Traumatic Brain Injury, Oncology, Infectious Diseases and Regenerative Medicine Stem Cell Applications
Press Release Source: Sangamo BioSciences, Inc. On Monday May 24, 2010, 7:00 am EDT
RICHMOND, Calif., May 24 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq:SGMO - News) announced today that data from research and preclinical programs focused on the development of zinc finger DNA-binding protein (ZFP) Therapeutics™ were described in fifteen presentations given by Sangamo scientists and collaborators at the 13th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The meeting was held in Washington, DC from May 17-22, 2010.
"Sangamo's ZFP technology provides an exciting new approach for the development of novel human therapeutics," said Luigi Naldini, M.D., Ph.D., Director of the San Raffaele Telethon Institute for Gene Therapy, Milan, and the principal investigator on several papers presented at the meeting. "ZFP nucleases (ZFNs) enable us to edit the human genome with unprecedented specificity and efficiency. This opens the way to correction of inherited mutations, a potentially revolutionary approach as it can restore a gene's function while preserving its natural regulation, likely overcoming the risks associated with random gene addition approaches. We are using this technology to investigate novel therapies for monogenic diseases and cancer."
The presentations made at the ASGCT meeting covered research and preclinical data from Sangamo's programs and collaborations in human therapeutics and technology development in primary human cells and stem cells. Therapeutic areas included ZFN-based approaches to infectious diseases such as HIV/AIDS and cytomegalovirus (CMV), monogenic diseases such as sickle cell anemia and epidermolysis bullosa, and oncology. Positive preclinical data were also presented from experiments using Sangamo's gene regulation technology to up-regulate the vascular endothelial growth factor-A (VEGF-A) gene in an animal model of traumatic brain injury. Philip Gregory, D. Phil., Sangamo's vice president of research and chief scientific officer, made a presentation entitled "Stem Cell Modification with Zinc Finger Nucleases," and chaired a session devoted to stem cell modification. All abstracts for the meeting are available online at http://www.asgct.org/am10/
"Sangamo's technology has the potential to revolutionize the field of cell and gene therapy," stated Barrie Carter, Ph.D., a member of Sangamo's scientific advisory board and the current President of ASGCT. "ZFN technology enables an efficient and precise process for making changes to the DNA sequence of a cell, which is necessary for therapeutic applications of gene-editing. ZFN-based therapeutics are already being evaluated by Sangamo in human clinical trials in HIV/AIDS and brain cancer. As this technology functions at the DNA level, it can be applied to address numerous diseases for which target genes have been identified."
"The data presented at this meeting highlight the breadth of potential applications for ZFP Therapeutics," said Dale Ando, M.D., Sangamo's vice president of therapeutic development and chief medical officer. "Our ZFP Therapeutic platform is maturing. We have completed four Phase 2 clinical trials, have an ongoing Phase 2b trial of our ZFP activator of VEGF-A, SB-509, in diabetic neuropathy and have three ongoing trials of our ZFN-based technology. At the same time, with our collaborators, we continue to define and develop new therapeutic opportunities."
Sangamo Biosciences, Inc. Q1 2010 Earnings Call Transcript
http://seekingalpha.com/article/202616-sangamo-biosciences-inc-q1-2010-earnings-call-transcript?source=sector_theme_sb_latest
Sangamo BioSciences Reports First Quarter 2010 Financial Results
Press Release Source: Sangamo BioSciences, Inc. On Monday May 3, 2010, 4:01 pm EDT
RICHMOND, Calif., May 3 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq:SGMO - News) today reported first quarter 2010 financial results and accomplishments.
For the first quarter ended March 31, 2010, Sangamo reported a consolidated net loss of $4.0 million, or $0.09 per share, compared to a net loss of $6.8 million, or $0.17 per share, for the same period in 2009. As of March 31, 2010, the company had cash, cash equivalents, marketable securities and interest receivable of $77.0 million.
Revenues for the first quarter of 2010 were $6.6 million, compared to $3.2 million for the same period in 2009. First quarter 2010 revenues were from the Company's collaboration agreements with Sigma-Aldrich Corporation and Dow AgroSciences, enabling technology agreements and research grants. The revenue recognized for the first quarter of 2010 consisted of $6.2 million in collaboration and enabling technology agreements and $449,000 in research grants. The increase in collaboration agreement revenues was primarily due to the license payment received from Sigma-Aldrich as part of its agreement with the Company which was expanded in the fourth quarter of 2009 and provides Sigma exclusive rights to develop and distribute zinc finger DNA-binding protein (ZFP)-modified cell lines for commercial production of protein pharmaceuticals and ZFP-engineered transgenic animals. The payment is being recognized as revenue ratably through July 2010, the remaining period of the Company's original research collaboration with Sigma-Aldrich.
Research and development expenses were $7.4 million for the first quarter of 2010, compared to $7.3 million for the same period in 2009. Research and development expenses for the first quarter of 2010 were primarily related to our clinical trials of SB-509 for diabetic neuropathy and SB-728-T for HIV/AIDS. General and administrative expenses were $3.3 million for the first quarter of 2010, compared to $2.9 million for the same period in 2009. The increase in general and administrative expenses was primarily due to increased personnel costs, including non-cash employee stock-based compensation, and professional fees.
Total operating expenses for the first quarter of 2010 were $10.7 million, compared to $10.2 million for the same period in 2009.
Net interest income and other income was $25,000 for the first quarter of 2010, compared to $193,000 for the same period in 2009. The decrease was due to lower interest rates on investments.
Recent Highlights
•Initiation of New Clinical Trials; Phase 2b study in Diabetic Neuropathy and Phase 1 Trial in Brain Cancer (Glioblastoma) with City of Hope: In January 2010, Sangamo initiated a Phase 2b trial of SB-509 in subjects with moderately severe diabetic neuropathy. The Juvenile Diabetes Research Foundation International (JDRF) also renewed its support for this program and will provide up to $3.0 million of funding for the trial. Sangamo's double blind, repeat-dosing, placebo-controlled Phase 2b study, SB-509-901, is designed to finalize dose, schedule and primary and secondary approvable endpoints for pivotal Phase 3 trials. Sangamo's collaborators at City of Hope also initiated a Phase 1 clinical trial of SB-313, a cytotoxic T-cell made glucocorticoid resistant using ZFN mediated gene-editing, that is being evaluated in subjects with recurrent or refractory glioblastoma.
•Presentation of Preliminary Data from Sangamo's Phase 1 Safety Trial of SB-728-T for HIV/AIDS: In January 2010, preliminary data from the University of Pennsylvania investigator sponsored Phase 1 safety study of Sangamo's zinc finger nuclease (ZFN) based product, SB-728-T, for HIV/AIDS were presented at the Keystone Symposium Session "HIV Biology and Pathogenesis." Sangamo's collaborator, Carl June, M.D., Director of Translational Research at the Abramson Family Cancer Research Institute at the University of Pennsylvania School of Medicine, presented the data as an invited speaker in an NIAID Workshop entitled "The Next Challenge: Elimination of HIV Reservoirs." Dr. June described data from a single HIV-positive subject treated with SB-728-T who, as part of the study, began a structured treatment interruption (STI) from his antiviral drug therapy after being treated with CCR5 negative ZFN-modified T-cells. During the monitoring period, the subject had stable levels of ZFN-modified T-cells which were well-tolerated and stable CD4+ T-cell counts. Interestingly, while the subject's viral load increased as expected during the STI period, the timing of the increase was somewhat delayed. Biopsy samples demonstrated that ZFN-modified cells appeared to circulate and traffic normally.
•Appointment of Two New Members of the Sangamo Board of Directors, Stephen G. Dilly, M.B.B.S, Ph.D. and William R. Ringo as Chairman of the Board: Dr. Dilly, who is currently President and CEO of APT Pharmaceuticals, Inc., a specialty drug company, was appointed to the board of directors effective March 31, 2010. Dr. Dilly has been closely associated with the development and launch of marketed drugs for many therapeutic areas in his previous senior executive positions in drug development at Chiron BioPharma, Genentech Inc. and SmithKline Beecham in the U.K. Mr. Ringo, who recently retired as Senior Vice President of Business Development, Strategy and Innovation at Pfizer Inc. where he had responsibility for guiding Pfizer's overall strategic planning and business development activities, was appointed as Chairman of the Sangamo Board of Directors on April 16, 2010.
Conference Call
Sangamo will host a conference call today at 5:00 p.m. ET, which will be open to the public. The call will also be webcast live and can be accessed via a link on the Sangamo BioSciences website in the Investor Relations section under "Events and Presentations" http://investor.sangamo.com/events.cfm. A replay of the webcast will also be available for two weeks after the call. During the conference call, the company will review these results, discuss other business matters, and provide guidance with respect to the rest of 2010.
The conference call dial-in numbers are 877-377-7553 for domestic callers and 678-894-3968 for international callers. The passcode for the call is 70476823. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 8:00 p.m. ET on May 3, 2010 to 11:59 p.m. ET on May 10, 2010. The conference call replay numbers for domestic and international callers are 800-642-1687 and 706-645-9291, respectively. The conference ID number for the replay is 70476823.
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http://www.sangamo.com/index.php
http://finance.yahoo.com/q/ks?s=SGMO+Key+Statistics
Sangamo Therapeutics, Inc., a clinical stage biopharmaceutical company, focuses on translating ground-breaking science into genomic therapies that transform patients' lives using platform technologies in genome editing, gene therapy, gene regulation, and cell therapy. The company?s proprietary zinc finger DNA-binding protein (ZFP) technology enables specific genome editing and gene regulation. The ZFPs could be engineered to make ZFP nucleases (ZFNs), proteins that could be used to specifically modify DNA sequences by adding or knocking out specific genes; and ZFP transcription factors (ZFP TFs), proteins that can be used to turn genes on or off. Its therapeutic products include SB-728-T, a ZFN-mediated autologous T-cell product for human immunodeficiency virus and acquired immunodeficiency syndrome (HIV/AIDS), which is in Phase II and Phase I clinical trials; and SB-728-HSPC that is in Phase I/II clinical trials for HIV/AIDS. The company also engages in Phase I/II studies of in vivo genome editing applications of ZFP Therapeutics for hemophilia B, Hemophilia A, and Mucopolysaccharidosis I (MPS) and MPS II, which are lysosomal storage disorder (LSD); proprietary preclinical programs in other LSDs; and research stage programs in certain central nervous system disorders and cancer immunotherapies. It has collaborative partnerships with Biogen Inc. to develop therapeutic genome editing products in hemoglobinopathies; and with Shire International GmbH to develop the preclinical development program in Huntington?s disease, as well as license agreement with Sigma-Aldrich Corporation to develop ZFP-based laboratory research reagents and Dow AgroSciences, LLC to modify the genomes or alter protein expression of plant cells, plants, or plant cell cultures. The company was formerly known as Sangamo BioSciences, Inc. and changed its name to Sangamo Therapeutics, Inc. in January 2017. Sangamo Therapeutics, Inc. was founded in 1995 and is headquartered in Richmond, California.
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