8 Dollar

Wohin, erneut nur über USD 8.00 oder USD 10.00 oder mehr, Fragen über Fragen....­

 

Kurs Aktie

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!" ...

 

Sangamo Ihr brauch Geduld, die Daten zu HIV/Ais sind sehr gut, aber auch in einem sehr frühen Stadium!
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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Vier Talks über eine Methode

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...

 

Sangamo, die nächste Dendreon? JMP "First clinical evidence of efficacy for Sangamo's ZFN platform demonstrat­es broad platform potential;­ reiterate Outperform­ &$12 target . Positive preclinica­l data shows stem cell applicabil­ity"
 
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  

update sangama

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:

SGMO

) 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."

 

und noch ein Bericht Sangamo BioScience­s Announces Four Data Presentati­ons At CROI 2011  Of Novel ZFN Therapeuti­c Approaches­ To The Treatment Of HIV/AIDS  

Main Category:

HIV / AIDS

Also Included In:

Conference­s

; 

Pharma Industry / Biotech Industry

Article Date: 04 Mar 2011 - 4:00 PST

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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

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)

 

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.

 

Source: Sangamo BioScience­s, Inc

 

 

news flut

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 :)

 

@elektromaniac

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

 

Die 8 Dollar wird wieder getestet

CUR 7.73

BID 7.75

ASK 8.43

 

anlauf

nimmt aber viel anlauf oder von 7,5 ? 8{

 

Wird mal Zeit!

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, die nächste Dendreon? Ich versteh das schon, Sangamo hat sich fast verdreifac­ht vom Tief und befindet sich in Phase I und Phase II Trials die viefverspr­echend sind, aber es wird noch Jahre dauern, wer durchhält und Glück hat bekommt einen Tenbagger!­

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.  

@electromaniac, Trendumkehr

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.

 

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.

 

ich ich denk bis august werden wir sicher die 10 euro sehen :-)  

ich wär schon froh

wenn mal die USD 10.00 erreicht würden.­...

 

Keine Frage

erreicht wird sie, hoffentlic­h aber nachhaltig­

 

Ja nee, is klar

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.

 

richtig 1000 punkte  

@94

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..

 

So noch mal für´n Masterofsalsa ich DENK bis august werden wir sicher die 10 euro sehen :-) des nächste mal einfach 2 mal lesen :-)  

10 Euro Frage

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

 

Neuen Jointventure rtreibt SGMO nach oben

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 Diseases  

Wow

SGMO vorboersli­ch schon auf 4.32....da­ kommt noch mehr!