Sangamo Treatment Shows Resistance To HIV In Study ( Seite 84)

Ich sehe das auch so. Bei hohem Verlust-Risiko - was sich aktuell durch den niedrigen Kurs relativiert - extrem hohe Chancen auf extreme Kurssteigerungen. Und die Zeit, bis Phase IIb greifbare Resultate liefert, ist überschaubar.

Antwort auf Beitrag Nr.: 41.704.331 von Reiki-Meister am 27.06.11 13:11:44Good news:


June 27, 2011

Sangamo BioSciences Announces Publication of First Demonstration of In Vivo Correction of Hemophilia Gene via Systemic Delivery of a ZFP Therapeutic®

Groundbreaking Nature Publication Demonstrates Direct In Vivo Correction of a Defective Gene and Highlights Potential Use of ZFN Technology for Treatment of Hemophilia and Other Monogenic and Rare Diseases





RICHMOND, Calif., June 27, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today the publication of a groundbreaking preclinical study demonstrating permanent functional correction of the gene that causes hemophilia B by the systemic delivery of zinc finger nucleases (ZFNs) . 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.

The work was carried out in the laboratory of Katherine High, M.D., Investigator, Howard Hughes Medical Institute, Professor of Pediatrics, University of Pennsylvania School of Medicine and Director, Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, in collaboration with Sangamo scientists and was published as an Advance Online Publication in Nature http://dx.doi.org/10.1038/nature10177.

"These data represent a significant advance in realizing efficient, systemic, therapeutic gene repair - the 'holy grail' of genetic medicine," said Dr. High, "With a single systemic administration of ZFNs and a donor sequence in a mouse model of hemophilia B, we demonstrated permanent correction of a defective human gene encoding the clotting factor, Factor IX, resulting in restoration of normal clotting times in the animal. ZFN-mediated gene-editing provides a new approach to monogenic disease and circumvents the problems of traditional gene-addition strategies that result in random insertion that may lead to malignancy or other unintended consequences. Genome editing also reinstates the wild-type sequence under the control of the endogenous regulatory sequences, assuring restoration of this critical aspect of normal gene expression. The study also demonstrated that permanent correction of the disease-related gene in situ can be achieved with therapeutically meaningful correction efficiencies."

"This is an important step forward in our goal to broaden the application of ZFN gene-editing via in vivo administration," stated Edward Lanphier, Sangamo's president and chief executive officer. "These data highlight the therapeutic potential of our ZFN technology and enable us to expand our ZFP Therapeutic pipeline to a growing number of monogenic and rare diseases."

The paper entitled "In vivo Genome Editing Restores Hemostasis in a Mouse Model of Hemophilia" described highly specific and efficient ZFN-mediated correction of a defective human Factor IX gene in a mouse model of hemophilia B by the delivery of ZFNs directly into animals. Stable levels of Factor IX protein made from the corrected human gene were measured in the plasma of the treated animals and resulted in the restoration of normal rates of blood clotting for the eight-month duration of the study. Moreover, the treatment was well tolerated as there were no deleterious effects on growth or liver function in the animals.

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. 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 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, the potential of ZFNs to treat human monogenic diseases such as hemophilia and other rare diseases, research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform. 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 assumes no obligation to update the forward-looking information contained in this press release.

SOURCE Sangamo BioSciences, Inc.

Hallo Sportsfreunde der Gentechnologie,
das scheint ja die Meldung zu sein, auf die Ihr solange gewartet habt.

Man spricht von bahnbrechenden Erfolgen!!!

Heisst im Umkehrschluß......heut muss das Teil durch die Decke fliegen. Eine andere Alternative gibt es dann nicht.

Ich freue mich für Euch und das meine ich ehrlich, zumal hier so ein Volltrottel geschrieben hatte, ich würde mich am Verlust anderer Börsenfrickler laben. Das ist natürlich geistiger Dünnpfiff, weil ich für den Einstiegspunkt anderer Börsis nichts kann. Es ist halt ein Volltrottel gewesen.... nicht mehr oder weniger.

Also....haltet durch und macht die Brocken klar....verdient habt Ihr es.

Und all denen, die immer das Wort Basher oder sonstige Anspielungen gebrauchen, sei gesagt:

Nicht jeder Börsi, der eine andere Meinung hat als Ihr, muss ein Basher sein.

Bis dahin und viel Glück

Hallo Oberst, danke für die netten Grüße. Musste vor 2 Monaten leider (ohne Verlust) rausgehen weil ich das Geld für das FA brauchte. Einer inneren Stimme folgend bin ich dann vor 2 Tagen mit den letzten Kröten wieder eingestiegen (ich weiss, so was macht man normalerweise nicht). Es gibt Dinge zwischen Himmel und Erde ...

Antwort auf Beitrag Nr.: 41.704.926 von Reiki-Meister am 27.06.11 15:03:19Vielleicht war das ein Wink mit dem Zaunpfahl....
Du müsstest ja jetzt einen besseren Einstiegskurs haben....das hat doch was

Antwort auf Beitrag Nr.: 41.704.926 von Reiki-Meister am 27.06.11 15:03:19Gratulation, den Schritt bereut man nicht.

Antwort auf Beitrag Nr.: 41.704.953 von Oberst-Crash am 27.06.11 15:08:10Die Rakete startet. Hoffentlich hat niemand of den Crashonkel gehoert.


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Sangamo BioSciences Announces Publication of First Demonstration of In Vivo Correction of Hemophilia Gene via Systemic Delivery of a ZFP Therapeutic® Groundbreaking Nature Publication Demonstrates Direct In Vivo Correction of a Defective Gene and Highlights Potential Use of ZFN Technology for Treatment of Hemophilia and Other Monogenic and Rare Diseases

PR Newswire

RICHMOND, Calif., June 27, 2011

RICHMOND, Calif., June 27, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today the publication of a groundbreaking preclinical study demonstrating permanent functional correction of the gene that causes hemophilia B by the systemic delivery of zinc finger nucleases (ZFNs) . 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.

The work was carried out in the laboratory of Katherine High, M.D., Investigator, Howard Hughes Medical Institute, Professor of Pediatrics, University of Pennsylvania School of Medicine and Director, Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, in collaboration with Sangamo scientists and was published as an Advance Online Publication in Nature http://dx.doi.org/10.1038/nature10177.

"These data represent a significant advance in realizing efficient, systemic, therapeutic gene repair - the 'holy grail' of genetic medicine," said Dr. High, "With a single systemic administration of ZFNs and a donor sequence in a mouse model of hemophilia B, we demonstrated permanent correction of a defective human gene encoding the clotting factor, Factor IX, resulting in restoration of normal clotting times in the animal. ZFN-mediated gene-editing provides a new approach to monogenic disease and circumvents the problems of traditional gene-addition strategies that result in random insertion that may lead to malignancy or other unintended consequences. Genome editing also reinstates the wild-type sequence under the control of the endogenous regulatory sequences, assuring restoration of this critical aspect of normal gene expression. The study also demonstrated that permanent correction of the disease-related gene in situ can be achieved with therapeutically meaningful correction efficiencies."

"This is an important step forward in our goal to broaden the application of ZFN gene-editing via in vivo administration," stated Edward Lanphier, Sangamo's president and chief executive officer. "These data highlight the therapeutic potential of our ZFN technology and enable us to expand our ZFP Therapeutic pipeline to a growing number of monogenic and rare diseases."

The paper entitled "In vivo Genome Editing Restores Hemostasis in a Mouse Model of Hemophilia" described highly specific and efficient ZFN-mediated correction of a defective human Factor IX gene in a mouse model of hemophilia B by the delivery of ZFNs directly into animals. Stable levels of Factor IX protein made from the corrected human gene were measured in the plasma of the treated animals and resulted in the restoration of normal rates of blood clotting for the eight-month duration of the study. Moreover, the treatment was well tolerated as there were no deleterious effects on growth or liver function in the animals.