Sangamo: Informationen zu Partner,
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wäre schon froh, wenn endlich mal die 6 die 5 vorm Komma dauerhaft ablösen würde... aber auch ich glaube, dass die Investition richtig war... irgendwann kommt der run... auf wieviel auch immer... ;-)
in diesem Sinne allen ein frohes "Kölle Alaaf!" ...
Die Phase II im Sommer wird entscheidend was der Kurs dann macht:
Sangamo BioSciences Announces Four Data Presentations at CROI 2011 of Novel ZFN Therapeutic Approaches to the Treatment of HIV/AIDS
Presentations 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
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{"s" : "sgmo","k" : "a00,a50,b00,b60,c10,g00,h00,l10,p20,t10,v00","o" : "","j" : ""} Press Release Source: Sangamo BioSciences, Inc. On Thursday March 3, 2011, 7:00 am
RICHMOND, Calif., March 3, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq:SGMO - News) announced that data from its programs to develop ZFN-based therapeutic approaches for the treatment of HIV/AIDS were discussed in four oral presentations at the 18th Conference on Retroviruses and Opportunistic Infections (CROI), held in Boston from February 27 to March 2, 2011.
"SB-728-T is a key addition to the toolbox of technologies required to move from the bone marrow transplant setting requiring a donor, to the use of the patient's own cells to replicate the 'functional cure' for HIV-infected patients in which one could create and maintain a reservoir of HIV-resistant immune cells," said Carl June, M.D., Director of Translational Research at the Abramson Family Cancer Research Institute at the University of Pennsylvania School of Medicine and an invited speaker at the conference. "The combination of robust and reproducible manufacturing, engraftment, trafficking, persistence and selective expansion of ZFN-CCR5-modified cells set this product apart from all other HIV therapies tested to date, and is an important step to move from the allogeneic bone marrow transplant setting to the use of the patient's own cells. In particular, the data showing selective expansion and enrichment of SB-728-T in the gut mucosa establishes mechanistic proof of concept that ZFN-CCR5-modified T-cells can constitute a compartment of the immune system that is protected from HIV infection."
In a symposium held on March 2, 2011, Dr. June presented preliminary data from the ongoing investigator-sponsored Phase 1 trial at the University of Pennsylvania as well as data from Sangamo's Phase 1 dose-escalation trial SB-728-902. The presentation included new data from subjects on highly active antiretroviral therapy (HAART) who underwent a treatment interruption (TI) for a specified period after treatment with SB-728-T as well as subjects on HAART with undetectable virus but suboptimal CD4+ T-cell counts (200-500 cells/ mm3).
Clinical Trial Data Summary
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 (TI) 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.
"Data presented this week at CROI 2011 establish the mechanistic 'proof of concept' of our ZFN-CCR5-modification in autologous T-cells and support broad development of this gene modification approach to HIV therapy," said Dale Ando, M.D., Sangamo's vice president of therapeutic development and chief medical officer. "Based on these very encouraging findings, we have recently extended our clinical studies to HIV-infected subjects who are not on HAART, or for whom HAART is no longer effective. This will enable us to comprehensively evaluate SB-728-T across the full spectrum of disease. We look forward to presenting data from all four patient segments later this year."
Abstract #165 Disruption of CCR5 in Zinc Finger Nuclease-treated CD4 T Cells: Phase I Trials (Presenter C. June)
Abstract #46 Successful and Persistent Engraftment of ZFN-M-R5-D Autologous CD4 T Cells (SB-728-T) in Aviremic HIV-infected Subjects on HAART (Presenter J. Lalezari)
Webcasts of all the presentations at CROI 2011 can be accessed via the following link http://www.retroconference.org/2011/data/files/webcast_2011.htm
Data Presented on Preclinical ZFN-Gene Modification Studies
A third presentation given by invited speaker, Paula Cannon, Ph.D., Associate Professor of Molecular Microbiology & Immunology at the Keck School of Medicine of the University of Southern California (USC), described data from Sangamo's preclinical program to develop CCR-specific ZFNs to modify the gene in hematopoietic stem cells (HSCs) to provide protection of the entire immune system from HIV infection. The work is part of a California Institute for Regenerative Medicine (CIRM) Disease Team Research Award which is funding IND-enabling studies of this approach. Dr. Cannon provided an update on progress in this program, demonstrating that the CCR5 gene of adult HSCs could be efficiently modified with ZFNs with high cell viability. In addition, the ZFN-CCR5-modified HSCs stably engraft in mouse models and retained the ability to develop into the multiple lineages of the immune system.
Abstract #164 CCR5 Knock-out in Hematopoietic Stem Cells (Presenter: P. Cannon)
Data were also presented from Sangamo's ZFN gene-modification program to generate CD4 T-cells that are resistant to so-called "X4-tropic" strains of HIV, strains of HIV that use the CXCR4 co-receptor to infect cells and are characteristic of late-stage HIV infections. This fourth presentation was given by Sangamo's collaborators in the laboratory of Robert Doms, M.D., Ph.D., Chair, Department of Microbiology, the University of Pennsylvania School of Medicine. Disruption of CXCR4 in CD4+ T cells avoids immune system toxicity associated with systemic disruption of the gene. The data demonstrated that ZFN modification of the CXCR4 gene in T-cells results in robust protection of CD4+ T cells from X4-tropic HIV challenge in vitro and confers protection from X4-tropic HIV in humanized mice. Future studies aim to combine CCR5- and CXCR4-ZFNs to totally eliminate HIV co-receptor expression to create completely HIV-resistant CD4+T cells.
Abstract # 47 Creating an HIV-resistant Immune System: Using CXCR4 ZFN to Edit the Human Genome (Presenter C. Wilen)
"Our highly specific gene-modification technology provides a novel platform for the development of transformational ZFP Therapeutics®," said Edward Lanphier, Sangamo's president and CEO. "As these presentations have demonstrated, Sangamo is rapidly advancing a broad pipeline of ZFN-based approaches to HIV/AIDS designed to address the spectrum of the disease. The ground-breaking clinical and preclinical data that we and our collaborators are generating validate our ZFN technology platform for development of novel therapeutic strategies for unmet medical needs, such as HIV and monogenic diseases. The processes and pathways that we have demonstrated in these programs are directly relevant to development of our future ZFP Therapeutics."
Dr. June has no financial relationship with Sangamo BioSciences, Inc.
About Sangamo
Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic® development program is currently in a Phase 2b clinical trial for evaluation of safety and clinical effect in patients with diabetic neuropathy and a Phase 2 trial in ALS. Sangamo also has a Phase 1 / 2 clinical trial and two ongoing Phase 1 clinical trials to evaluate the safety and efficacy for the treatment of HIV/AIDS as well as a Phase 1 trial for the treatment for recurrent glioblastoma multiforme. Other therapeutic programs are focused on Parkinson's disease, monogenic diseases, neuropathic pain and nerve regeneration. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for gene modification. Sangamo has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website at http://www.sangamo.com/.
ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.
This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references relating to research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform for the treatment of HIV/AIDS, including a potential functional cure of HIV/AIDS, the expansion of clinical studies for HIV-infected individuals and the initiation of additional preclinical studies of ZFN-gene modification. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.
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das ist doch was. Und das von unterschiedlichen Gruppen. Da gibt es sicher Erwartungen, die sich im Kurs widerspiegeln. Und zwar vor den Ergebnissen im Sommer. Außerdem hat Sangamo noch mehr in peto...
JMP SECURITIES
Biotechnology – Update
Sangamo BioSciences, Inc. (1)
Beyond Technical Proof of Concept to Evidence of Efficacy in HIV with More to Come
MARKET OUTPERFORM
Target Price $12.00
INVESTMENT HIGHLIGHTS
· First clinical evidence of efficacy for Sangamo's ZFN platform demonstrates broad platform potential; reiterate Market Outperform rating and $12 price target. In our view, data presented this week at the Conference on Retroviruses and Opportunistic Infections (CROI) for Sangamo's novel HIV/AIDS development candidate, SB-728, exceeded our expectations, validating the technology and furthermore providing preliminary evidence of efficacy for a potentially paradigmshifting HIV treatment. While the patient numbers remain limited, we believe the clinically meaningful and persistent responses observed in these initial trials enhance our conviction that clinical proof-of-concept with ZFN-edited T-cells can be demonstrated in ongoing trials for SB-728. Additionally, we view these data as important in highlighting the future of the ZFN technology broadly in the developing fields of gene therapy for selected orphan diseases. Our $12 price target is based on a sum-of-the-parts analysis of Sangamo’s hybrid business, including $4 per share for industrial uses, approximately $7 for current therapeutic candidates and IP, and ~$1 in FYE11 cash.
· Strong platform to build on with further value-driving catalysts throughout 2011. Additional data from the HIV/AIDS program, including patients who are not on HAART or not responsive to HAART, are expected to be presented later in 2011. Most important, we believe, is the continued validation of the ZFN platform, which now includes early-stage results in humans in addition to the marketed products in rodents and in cell lines. In all the attention being given to the headline HIV/AIDS results, which we view as impressive, we believe investors should not lose sight of the
positive preclinical results shown at ASH in a model of hemophilia. Finally, results from the Phase IIb trial in diabetic neuropathy are on track to read out in 4Q11, although we see this therapeutic setting as less compelling than the adoptive immunotherapy setting with modified T-cells. Based on our ongoing diligence, we continue to be optimistic for the prospect of additional clinical validation for Sangamo's pipeline going forward (Figure 6).
· Mechanistic rationale for ZFN-driven T-cell response presented by key opinion leaders. Data presented on Monday detailed six aviremic HIV/AIDS patients from the Quest/UCLA study who were on HAART treatment but with persistently low CD4+ counts (<500 cells/ul). The patients were treated with their own T-cells, which had been removed, enriched for CD4+ positive cells, and modified with the SB-728 ZFN designed to disrupt the CCR5 gene, as outlined in Figure 1. All six of the patients had significant and persistent engraftment of the modified T-cells, and five of six had significant and persistent improvement in CD4+ T-cell counts and the CD4:CD8 ratio. A KOL stated that these CD4+ cell count results represent a "greater improvement" than any prior adoptive T-cell
treatment. The second of Monday's presentations demonstrated deletion of the other HIV receptor, CCR4, in human CD4+ T-cells and the ability of these cells to avoid HIV infection in vitro and in mice. A future goal is to delete both receptors (CCR5 and CCR4) from CD4+ T-cells to enable more robust anti-HIV efficacy.
· Broader scope highlighted by multiple patient populations. In yesterday's afternoon session at CROI, Professor Carl June from The University of Pennsylvania presented a clinical summary of Sangamo's ongoing HIV/AIDS trials as well as additional patient results from investigator sponsored trials. Data were presented from three patients who are part of the U-Penn structured treatment interruption (STI) trial. All three patients were aviremic; two had CD4+ counts above 450 and one below 500; and all were on HAART treatment. As part of the STI, the patients with an initial CD4+>450 underwent a twelve-week treatment halt one month after being infused with SB- 728-T. These patients in particular demonstrated some interesting viral dynamics that the company believes may be informative regarding the function of SB-728-T (Figure 2). As shown in Figures 3 and 4, the two STI patients (patients 201 and 203) demonstrated persistent engraftment and trafficking of modified CD4+ T-cells, while the patient with initial CD4+<500 (patient 306) showed strong transient responses, with longer-term results pending. Data from all nine patients, including the six presented Monday, showed that modified CD4+ cells persistently engrafted at a median level of 5.2% of all CD4+ cells at day 90 following infusion. In all nine patients there were no observed SAEs and a few moderate AEs, which were transient in nature.
· Positive preclinical data shows stem cell applicability. Professor Paula Cannon from UCLA presented results from studies examining CCR5-knockout human hematopoietic stem cells obtained from cord blood. She presented results demonstrating the engraftment of these cells in mice lacking an immune system. Convincing results of the ability of these cells to resist and potentially eliminate HIV from the blood and gut mucosa are shown in Figure 5. The presentation provided a forward looking perspective for the ZFN program's applicability to a stem cell population, which can be permanently engrafted. Since stem cells can give rise to many important cell types, such as T-cells, macrophages, B-cells and many others, this program has wide-ranging potential for advanced treatments.
FIGURE 1: SB-728-T Treatment Protocol
Source: CROI presentation
FIGURE 2: Viral Responses to Treatment Interruption
Source: CROI presentation
FIGURE 3: CCR5-Modified CD4+ T-Cell Persistance
Source: CROI presentation
FIGURE 4: Trafficking and Engraftment of SB-728-T to Mucosa
LOD = limit of detection
Source: CROI presentation
FIGURE 5: Control of HIV in ZFN-Modified HSCs
Source: CROI presentation.
FIGURE 6: Sangamo's Pipeline
Therapeutic Area Program ID
Diabetic Neuropathy SB-509
ALS SB-509
HIV/AIDS SB-728
Glioblastoma SB-313
Parkinson's Preclinical
Neuropathic Pain Preclinical
Spinal Cord Injury Preclinical
Monogenic and Rare Diseases Preclinical
Development Phase
Preclinical Phase I Phase II Phase III
Source: Company reports
INVESTMENT RISKS
Given that Sangamo is still in the development stage, the primary risk to our investment thesis involves the ability of it to progress its development programs into later-stage trials. In addition and in relation to its internal research programs, other risks to our investment thesis involve being able to successfully progress these programs into clinical trials and subsequently into later-stage trials. The last risk we would point out is the company's ability to continue to fund operations through product commercialization.
COMPANY DESCRIPTION
Richmond, California-based Sangamo BioSciences, Inc. uses its proprietary zinc finger DNA-binding protein (ZFP) technology to develop gene therapy treatments for a number of diseases. The company's lead therapeutic, SB-509, is a ZFP that activates the VEGF-A gene in order to promote blood vessel growth, nerve regeneration, and neuroprotection. Sangamo has multiple clinical and preclinical programs with SB-509 in several vascular and neurodegenerative diseases, including diabetic neuropathy, peripheral arterial disease, critical leg ischemia, amyotrophic lateral sclerosis, and spinal cord injury. Diabetic neuropathy is the leading indication in Phase II development. Other early-stage programs include ZFN CCR5 for the treatment of HIV and IL-13 zetakine for glioblastoma.
FIGURE 7: Sangamo BioSciences Earnings Model ($000s, except per share data)
FY06A FY07A FY08A FY09A Mar-10A Jun-10A Sep-10A Dec-10A FY10A Mar-11E Jun-11E Sep-11E Dec-11E FY11E FY12E
Industrial revenue
Sublicensing & royalty revenues - - - - - 1,000 1,500 2,500 7,500
Milestones - -
Total industial revenue - - - - - - - 1,000 1,500 2,500 7,500
Grants/Collaboration revenue 7,885 9,098 16,186 22,187 6,648 6,525 2,943 4,689 20,805 3,375 4,375 4,875 5,875 18,500 20,200
Pharmaceutical revenue
Partnered therapeutic milestones - 15,000 15,000 25,000
Proprietary therapeutic revenue - - 25,000
anliegend ein neuer Bericht zu Sangamo v. 03.03.11
Sangamo (SGMO) Updates on ZFN-Based Treatments for HIV/AIDS
March 3, 2011 7:14 AM EST
Sangamo BioSciences, Inc. (Nasdaq:
) presents data for four ZFN-based therapeutic approaches for the treatment of HIV/AIDS.
From the release:
"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 (TI) 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."
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Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced that data from its programs to develop ZFN-based therapeutic approaches for the treatment of HIV/AIDS were discussed in four oral presentations at the 18th Conference on Retroviruses and Opportunistic Infections (CROI), held in Boston from February 27 to March 2, 2011.
"SB-728-T is a key addition to the toolbox of technologies required to move from the bone marrow transplant setting requiring a donor, to the use of the patient's own cells to replicate the 'functional cure' for HIV-infected patients in which one could create and maintain a reservoir of HIV-resistant immune cells," said Carl June, M.D., Director of Translational Research at the Abramson Family Cancer Research Institute at the University of Pennsylvania School of Medicine and an invited speaker at the conference. "The combination of robust and reproducible manufacturing, engraftment, trafficking, persistence and selective expansion of ZFN-CCR5-modified cells set this product apart from all other
therapies tested to date, and is an important step to move from the allogeneic bone marrow transplant setting to the use of the patient's own cells. In particular, the data showing selective expansion and enrichment of SB-728-T in the gut mucosa establishes mechanistic proof of concept that ZFN-CCR5-modified T-cells can constitute a compartment of the immune system that is protected from HIV infection."
In a symposium held on March 2, 2011, Dr. June presented preliminary data from the ongoing investigator-sponsored Phase 1 trial at the University of Pennsylvania as well as data from Sangamo's Phase 1 dose-escalation trial SB-728-902. The presentation included new data from subjects on highly active antiretroviral therapy (HAART) who underwent a treatment interruption (TI) for a specified period after treatment with SB-728-T as well as subjects on HAART with undetectable virus but suboptimal CD4+ T-cell counts (200-500 cells/ mm3).
Clinical Trial Data Summary
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 (TI) 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.
"Data presented this week at CROI 2011 establish the mechanistic 'proof of concept' of our ZFN-CCR5-modification in autologous T-cells and support broad development of this gene modification approach to HIV therapy," said Dale Ando, M.D., Sangamo's vice president of therapeutic development and chief medical officer. "Based on these very encouraging findings, we have recently extended our clinical studies to HIV-infected subjects who are not on HAART, or for whom HAART is no longer effective. This will enable us to comprehensively evaluate SB-728-T across the full spectrum of disease. We look forward to presenting data from all four patient segments later this year."
Abstract #165 Disruption of CCR5 in Zinc Finger Nuclease-treated CD4 T Cells: Phase I Trials (Presenter C. June)
Abstract #46 Successful and Persistent Engraftment of ZFN-M-R5-D Autologous CD4 T Cells (SB-728-T) in Aviremic HIV-infected Subjects on HAART (Presenter J. Lalezari)
Data Presented on Preclinical ZFN-Gene Modification Studies
A third presentation given by invited speaker, Paula Cannon, Ph.D., Associate Professor of Molecular Microbiology & Immunology at the Keck School of Medicine of the University of Southern California (USC), described data from Sangamo's preclinical program to develop CCR-specific ZFNs to modify the gene in hematopoietic
(HSCs) to provide protection of the entire immune system from HIV infection. The work is part of a California Institute for Regenerative Medicine (CIRM) Disease Team Research Award which is funding IND-enabling studies of this approach. Dr. Cannon provided an update on progress in this program, demonstrating that the CCR5 gene of adult HSCs could be efficiently modified with ZFNs with high cell viability. In addition, the ZFN-CCR5-modified HSCs stably engraft in mouse models and retained the ability to develop into the multiple lineages of the immune system.
Abstract #164 CCR5 Knock-out in Hematopoietic Stem Cells (Presenter: P. Cannon)
Data were also presented from Sangamo's ZFN gene-modification program to generate CD4 T-cells that are resistant to so-called "X4-tropic" strains of HIV, strains of HIV that use the CXCR4 co-receptor to infect cells and are characteristic of late-stage HIV infections. This fourth presentation was given by Sangamo's collaborators in the laboratory of Robert Doms, M.D., Ph.D., Chair, Department of Microbiology, the University of Pennsylvania School of Medicine. Disruption of CXCR4 in CD4+ T cells avoids immune system toxicity associated with systemic disruption of the gene. The data demonstrated that ZFN modification of the CXCR4 gene in T-cells results in robust protection of CD4+ T cells from X4-tropic HIV challenge in vitro and confers protection from X4-tropic HIV in humanized mice. Future studies aim to combine CCR5- and CXCR4-ZFNs to totally eliminate HIV co-receptor expression to create completely HIV-resistant CD4+T cells.
Abstract # 47 Creating an HIV-resistant Immune System: Using CXCR4 ZFN to Edit the Human Genome (Presenter C. Wilen)
"Our highly specific gene-modification technology provides a novel platform for the development of transformational ZFP Therapeutics®," said Edward Lanphier, Sangamo's president and CEO. "As these presentations have demonstrated, Sangamo is rapidly advancing a broad pipeline of ZFN-based approaches to HIV/AIDS designed to address the spectrum of the disease. The ground-breaking clinical and preclinical data that we and our collaborators are generating validate our ZFN technology platform for development of novel therapeutic strategies for unmet medical needs, such as HIV and monogenic diseases. The processes and pathways that we have demonstrated in these programs are directly relevant to development of our future ZFP Therapeutics."
Dr. June has no financial relationship with Sangamo BioSciences, Inc.
Source: Sangamo BioSciences, Inc
diese informationsflut ist mal was schönes werden hoffentlich viele aufmerksam auf die aktie :)
aber es ist echt schwer bei den vielen fachausdrücken und abkürzungen das wichtige herauszufiltern :) besonders für techniker die eher nach keep it small & simple arbeiten :)
bin der gleichen Meinung. Ich versuche deswegen bei langen Berichten nur das (für mich Interessante) rauszukopieren, um somit dem Leser das Wesentliche mitzuteilen. Dann gebe ich noch meinen Senf dazu, und natürlich die Quelle, falls ich sie nicht vergesse.
Eigentlich wäre es mir lieber, wenn in den Medien mal estwas mehr Ruhe einkehren würde. Eventuell würde es der Aktie sogar Auftrieb geben. Bei Paion war es doch ähnlich. Ende letzten Jahres gute News, seither gings runter, bis vor wenigen Tagen jedenfalls. Und nun gehts wieder bergauf, ohne News.
Und bei Evotec? Das gleiche Drama. Viele pos. News kamen, die Aktien geht in den Keller. Hoffe, die findet ebenfalls bald wieder Anschluss an ältere Höchststände...
Sorry für den kleinen Exkurs
Wünsche nen guten Start in die ausklingende Karnevalswoche,
ffa
Hoffe, dass die Aktie endlich mal über 8 Dollar steigt und auch bei 8 bleibt! Ich versteh nicht, warum der Titel so gefallen ist...
Grüsse
Sangamo: The Next Pinnacle in HIV Treatment?
by: Prohost Biotech March 08, 2011 | about: SGMO Font Size: PrintEmail Recommend 0 Share this page
Share0 When the human immunodeficiency virus (HIV) was identified as the cause of the mysterious immune system depletion characterized by horrific vulnerability to infection, multiple dreadful symptoms and ultimately death, the world panicked. The medical and research communities were taken by surprise. Nothing could be said or done to help the agonizing victims or prevent their imminent death. It took a few years to introduce the first HIV drug, a nucleoside reverse transcriptase inhibitor (NRTI). This milestone was followed by the development and marketing of non-nucleoside reverse transcriptase inhibitors (NNRTI), then a third class antiretroviral protease inhibitors (PIs). As HIV continued to reproduce and resistant variants of the virus emerged, It became necessary to combine the antiretroviral drugs in what has become known as highly active antiretroviral therapy (HAART). Indeed, the HAART combination resulted in profound reductions in AIDS morbidity and mortality.
The Increased familiarity and experience with the HIV and with the HIV drugs led to the development of antiretroviral drugs that could be taken once or twice daily as well as combinations of drugs in one tablet. These improvements resulted in easier use, hence, improved compliance and increased survival. Studies aiming at further improving HIV treatments never stopped. Recently, one of the studies demonstrated that in asymptomatic patients with higher CD4+ T cell counts, early initiation of HAART further improves survival.
Despite the promises of HAART, HIV treatment, nevertheless, is still fraught with problems. Toxicities arising from long-term use, high potential for drug interactions, and viral resistance still pose threats to many patients. Recognizing the fact that the approved drugs do not eradicate HIV infection, but merely suppress viral replication, sometimes to levels undetectable, necessitated new approaches that would increase treatment options. Blocking HIV entry into lymphocytes and macrophages became a priority target. However, the ideal options have been regimens that would induce the host immune system to eradicate HIV, or to increase cells’ resistance to the virus.
Scientists’ hopes of preventing HIV from entering host cells were realized first with the development of the drug, enfuvirtide, which binds to an HIV envelope glycoprotein, preventing the virus from fusing to the external surface of the host cell. The discovery that HIV attaches to host cell receptors (co-receptors), CCR5 and CXCR4 and that these transmembrane G protein-coupled receptors are critical for HIV entry into the cells, led to the discovery and development of therapeutics that would block CCR5, thereby prevent HIV from gaining access to the cells. Maraviroc, which binds to CCR5, was the first entry inhibitor to be developed with this mechanism of action. The approvals of enfuvirtide and maraviroc have provided additional treatment options for HIV infected patients. Still, sustained treatment with these drugs is required to prevent reactivation of the virus and disease progression, which subjects the patients to adverse effects and the drugs to viral resistance.
What’s next?
It seemed that learning from Nature might prove to bring a near answer to the HIV tragedy. A population of individuals who demonstrated immunity to HIV infection despite multiple exposures to the virus has been identified. These individuals have been found to have a natural mutation, CCR5delta32, resulting in the expression of a shortened (truncated) and non-functional CCR5 protein. Scientists also found out that mutation in one of the two copies of the CCR5 gene kept HIV in check without drugs when the carriers of this one copy mutation contracted the virus.
In December 2010 the journal Blood reported that an AIDS patient who had leukemia received a bone marrow transplant from a “matched” donor with this delta-32 CCR5 mutation. Transfer of the hematopoietic stem cells from the bone marrow of the delta-32 donor provided a self-renewable and potentially lifelong source of HIV-resistant immune cells in the recipient. To the specialists’ surprise, when the patient discontinued all antiretroviral drugs, CD4 counts increased and viral load dropped to an undetectable level.
SANGAMO
These true stories opened the biotechnology firm Sangamo’s (SGMO) eyes. The firm realized that its technology would enable it to mimic Nature. This firm was established around a technology based on engineering proteins that nature uses to control gene expression in living organisms from yeast to humans. The engineered proteins (DNA transcription factors), which are known as finger DNA-binding proteins (ZFPs) have two domains: the first is the ZFP portion that recognizes and specifically binds to a particular DNA sequence and the second is a functional domain. When the functional domain is brought into close proximity with a gene, it induces biologic effects that vary from activation, to repression, modification, correction, disruption, and addition.
Indeed, Sangamo used its ZFN-mediated editing technology to replicate the naturally occurring human mutation in CCR5 that renders individuals largely resistant to infection with the most common strain of HIV. Sangamo’s approach comprised removal of CD4+ T-cells from the blood of immunologic non-responders HIV-infected subjects who are currently on highly active antiretroviral therapy (HAART), having undetectable levels of virus but suboptimal CD4+ T-cell counts. These CD4+ T-cells were then treated with Sangamo’s ZFNs that modify the DNA sequence encoding the CCR5 gene. The process has, indeed, generated CCR5-modified, autologous T-cell product (SB-728-T), which was infused in HIV/AIDS patients.
The data were stunning. The approach proved to be safe, as infusion of SB-728-T was well tolerated. The CCR5-modified cells successfully engrafted in all subjects and resulted in a durable improvement in total CD4+ T-cell counts in five of six of the subjects analyzed. Five of the six subjects exhibited sustained improvements in their CD4:CD8 T-cell ratio, which is an indicator of immunologic health. The ZFN-CCR5-modified cells exhibited normal T-cell growth kinetics and trafficking and underwent selective expansion in the gut mucosa, a major reservoir of virus in the body, suggesting that the cells were resistant to HIV infection.
Aren’t these the first steps on the road to developing an HIV/AIDS "functional cure"?
Providing AIDS patients with a protected CD4+ T-cell population resistant to HIV infection has been the dream that had yet to come true. This dream seems to materialize at Sangamo’s hands, as the data from experimentation were very encouraging. Dale Ando, M.D, Sangamo's vice president of therapeutic development and chief medical officer said it all when he stated: “The data represent an early validation of the feasibility of Sangamo’s gene modification approach for the treatment of HIV/AIDS.” “We have confirmed that we have a scalable manufacturing process and that from a single apheresis we can manufacture doses of SB-728-T of 10 to 30 billion cells. We have also learned that a single infusion of these cells is well tolerated and that the cells engraft, multiply and persist in the body. The modified cells behave like unmodified cells and traffic to the gut mucosa. In addition, we observed selective expansion of ZFN-modified cells in the gut mucosa, a major reservoir of HIV in the body, suggesting that these cells are protected from HIV and selectively enrich in the presence of the virus. The data also underscore our decision to expand our clinical studies to populations of HIV-infected individuals that have active viral replication and thus higher viral loads and retain immune reactivity to HIV. From such studies we will be able to assess the effect of ZFN-CCR5-modified T- cells on overall T-cell numbers and the course of the viral infection."
For more data go to: http://investor.sangamo.com/releasedetail.cfm?ReleaseID=554198
What do we think?
We think what has been realized is a no less than a breathtaking breakthrough. The data are preliminary, but solid and unprecedented. Besides the hope they gave HIV victims, they offered proof of and validation of Sangamo’s Zink finger technology. We sincerely believe that this technology is on its way to offering the first wave of cures for many diseases that have yet to find a safe and effective treatment.
Disclosure: Long Sangamo.
seit Tagen startet sgmo realtiv stark am Morgen und endet schwaecher am Abend (Nasdaq). Gestern war es umgekehrt.
Vielleicht gibt es ja endlich eine Trendumkehr.
Das Tal der Traenen scheint ueberschritten zu sein. Nach den vielen guten Nachrichten, ist diese nun auch faellig. Wuerde mich wundern, falls 2011 die 10 Euro nicht sehen waeren.
Hallo,
"bis august werden wir sicher die 10 euro sehen", "keine Frage, erreicht wird sie"
30 Prozent Plus bis August? Toll. Und das ganz sicher? Noch toller. Na, dann sollten alle Wissenden alles was sie haben ganz schnell zu Geld machen, dazu noch einen Kredit aufnehmen und alles in Sangamo stecken. Das wird ja ein Riesengeschäft, noch dazu risikolos.
Leider bin ich zu doof, meine Glaskugel richtig zu bedienen. Ich würd auch sooo gern im vorraus wissen, wie sich Aktienkurse entwickeln. Meine Kugel vergisst nur immer, mich rechtzeitig vor Kriegen, Revolutionen, Erdbeben, Zunamis und ähnlichen Dingen zu warnen.
Zum Glück weiß ich ja jetzt, daß bis August nur noch Friede Freude Eierkuchen ist.
es hat ja niemand behauptet dass an einem bestimmten tag der kurs von 10,- erreicht wird, nur BIS august, u das ist immerhin gut möglich bei einer solch volatilen aktie; also verdreh hier nicht die tatsachen und versuch hier nicht klugscheißerisch andere zu belehren.. also echt..
Mit einer kleinen Einschraenkung: falls miese Nachrichten kommen oder die Weltwirtschaft massiv einbricht, wird es kaum was mit der Marke werden. Beide Szenarien erwarte ich allerdings nicht in diesem Jahr
Shire bekommt Rechte von Sangamo, die ZFP Methode in ihrer Forschung anwenden zu duerfen. Das duerfte noch fuer weiteren Anstieg sorgen.
Quelle: FinanzNachrichten.de
Shire and Sangamo Collaborate to Develop Therapeutics for the Treatment of Hemophilia and Other Monogenic DiseasesDUBLINand RICHMOND, Calif., Feb. 1, 2012 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, and Sangamo BioSciences, Inc. (NASDAQ: SGMO), a leader in genome-editing technology, announced today that they have entered into a collaboration and license agreement to develop therapeutics for hemophilia and other monogenic diseases based on Sangamo's zinc finger DNA-binding protein (ZFP) technology.
Shire will receive exclusive world-wide rights to ZFP Therapeutics® designed to target four genes (for blood clotting Factors VII, VIII, IX and X) which will be used to investigate curative therapies for hemophilia A and B. Shire also receives the right to designate three additional gene targets. Sangamo is responsible for all activities through submission of Investigational New Drug (IND) Applications and European Clinical Trial Applications (CTA) for each product and Shire will reimburse Sangamo for its internal and external research program-related costs. Shire is responsible for clinical development and commercialization of products arising from the alliance. Shire will pay Sangamo $13 million upfront followed by research, regulatory, development and commercial milestone payments, and royalties on product sales.
"Sangamo's ground-breaking ZFP gene-editing technology will enable us to expand our therapeutic pipeline into therapies for other genetic disorders such as hemophilia," said Sylvie Gregoire, president of Shire's Human Genetic Therapies business. "While still early in the clinical development process, this DNA-binding protein technology is aligned with our focus of developing new treatments that can add value for physicians, patients and their families, and the healthcare community overall."
"We are delighted to be partnering the first of our monogenic disease programs with Shire, a company known for its development of innovative medicines for genetic diseases," said Edward Lanphier, Sangamo's president and chief executive officer. "This alliance is further validation of our ZFP platform as a transformative technology for the development of novel therapeutics, which have the potential to revolutionize the treatment of a wide range of genetic diseases."
Sangamo's ZFP Therapeutic approach utilizes its proprietary ZFP nuclease (ZFN) and ZFP transcription factor (ZFP TF) technology. ZFPs can be engineered to recognize any specific DNA sequence within a gene, and may be applicable to certain Shire therapeutic areas, including hematology and lysosomal storage disorders.