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Werkstatt für unser Erbgut
von Sascha Karberg
Nach herben Rückschlägen schöpfen Gentherapeuten wieder Hoffnung. Neue Methoden sollen helfen, defekte Gene zu reparieren.
Prinzip der DNA-AnalyseNimmt man ihre Berufsbezeichnung wörtlich, müssten Gentherapeuten eigentlich Gene behandeln. Doch obwohl seit fast 15 Jahren Tausende von Gentherapien getestet wurden, war es bisher unmöglich, auch nur ein defektes Gen in einer menschlichen Zelle gezielt zu reparieren. Bestenfalls eine intakte Version des mutierten Gens ließ sich ins Erbgut einschleusen. Erst heute berichten Forscher der kalifornischen Biotech-Firma Sangamo im Fachmagazin "Nature" von der ersten erfolgreichen Reparatur eines menschlichen Gens mit Hilfe eines neu entwickelten molekularen Werkzeugs. Und weisen der Gentherapie damit einen Ausweg aus der Krise, in der sie nach diversen Fehlschlägen und sogar tragischen Todesfällen steckt.
Es ist sicherlich kein Zufall, dass das Team um Michael Holmes für den Test des neuen Werkzeugs ausgerechnet die Erbkrankheit wählte, mit der die Euphorie um die Gentherapie begann: Am 14. September 1990 präsentierten US-Forscher die damals vierjährige Ashanti da Silva, deren Immunsystem durch ein mutiertes Gen lahm gelegt war.
SCID (Severe Combined Immunodeficiency) heißt die Krankheit, die sonst harmlose Keime zur tödlichen Bedrohung für Patienten macht. Die Forscher entnahmen dem Mädchen blutbildende Zellen und schleusten intakte Kopien des Gens ein. Und tatsächlich wuchsen genügend Immunzellen heran. Ashanti, vorher hermetisch von der Außenwelt abgeschirmt, konnte fortan ohne Angst vor Infektionen auf den Spielplatz.
Noch kein glücklicher Patient
Einen glücklichen Patienten kann Michael Holmes, Leiter der Studie und Forschungsdirektor bei Sangamo, leider noch nicht präsentieren. Er hat die neue Methode bisher nur an menschlichen Zellen in der Petrischale getestet. Die Wissenschaftler sind vorsichtiger geworden, seit die erste Euphorie verflogen ist und die ersten technischen Probleme aufgetreten sind. Unvergessen ist der Fall des 18-jährigen Jesse Gelsinger, der bei einem Gentherapie-Versuch starb. Unvergessen sind auch die drei SCID-kranken Kinder, die in Paris zunächst erfolgreich mit einer Gentherapie behandelt wurden, später aber an Blutkrebs erkrankten - ein Kind starb inzwischen.
Denn das ist die größte Gefahr beim Einschleusen von künstlicher DNA: Nachgebaute Gene können offenbar Krebsgene aktivieren, weil sie zufällig ins Erbgut eingebaut werden und dabei wichtige Erbinformationen zerstören.
Diese Gefahr lässt sich mit dem neuen Werkzeug vermeiden, da es aus zwei Komponenten besteht. Der so genannte Zinkfinger ist ein Protein, das eine bestimmte Abfolge der DNA-Bausteine erkennt. So lässt sich jede gewünschte Stelle im Erbgut ansteuern. Dann wird die zweite Komponente aktiv, eine Art molekulare Schere, die DNA schneidet. Durch den Schnitt wird ein natürlicher Reparaturmechanismus ausgelöst, bei dem die losen DNA-Enden nicht nur zusammengeführt, sondern auch die DNA-Bausteine vor und hinter dem Schnitt ersetzt werden. Damit die richtige und nicht wieder die mutierte Abfolge von DNA-Bausteinen ergänzt wird, stellen die Wissenschaftler außer ihrem Werkzeug auch noch ein Stück DNA mit der korrekten Gensequenz als Korrekturvorlage zur Verfügung.
Der "Quantensprung" der Sangamo-Forscher
Einen "Quantensprung" nennt Albert Jeltsch von der International University Bremen die Arbeit der Sangamo-Forscher neidlos. Der Molekularbiologe bastelt ähnliche Werkzeuge, indem er an den DNA-bindenden Zinkfinger eine Komponente hängt, die die DNA nicht schneidet, sondern nur verändert. Jeltsch hofft, Gene des Herpes-Virus, das beim Menschen Herpesbläschen verursacht, stillzulegen.
Sangamo will die Zinkfinger-Technologie vor allem zur Therapie von Erbkrankheiten einsetzen, die Blutzellen betreffen, wie zum Beispiel Sichelzellenanämie. Außerdem entwickelt das Unternehmen nach eigenen Angaben eine Therapie gegen HIV-Infektion. Dabei soll das Werkzeug in den Blutzellen ein Gen zerstören, das die Aids-Viren für die Infektion der T-Zellen benötigen.
Aaron Klug, Nobelpreisträger und Entdecker der Zinkfinger-Proteine aus dem britischen Cambridge, ist schon jetzt von den neuen Perspektiven begeistert: "Die Arbeit ist ein Meilenstein auf dem Weg zu genverändernden Therapien."
--------------------------------------------------------------------------------
Variantenreiche Wissenschaft
Herkömmlich Die intakte Version eines mutierten Gens wird ins Erbgut eingeschleust. Noch gibt es eine Reihe ungelöster technischer Probleme.
Neu Die Forscher lösen im Erbgut einen natürlichen Reparaturmechanismus aus. Dazu haben sie ein Werkzeug entwickelt, das sich aus einem speziellen Protein, dem so genannten Zinkfinger, und einer Art molekularen Schere zusammensetzt, mit der sich DNA schneiden lässt.
ftd.de, 03. 04. 2005
© 2005 Financial Times Deutschland, © Illustration: FTD/am; Quelle: Mark Benecke
Hier der Chart
Homepage
http://www.sangamo.com/news/news.php
Eine interessante Anlage
gruss meislo
von Sascha Karberg
Nach herben Rückschlägen schöpfen Gentherapeuten wieder Hoffnung. Neue Methoden sollen helfen, defekte Gene zu reparieren.
Prinzip der DNA-AnalyseNimmt man ihre Berufsbezeichnung wörtlich, müssten Gentherapeuten eigentlich Gene behandeln. Doch obwohl seit fast 15 Jahren Tausende von Gentherapien getestet wurden, war es bisher unmöglich, auch nur ein defektes Gen in einer menschlichen Zelle gezielt zu reparieren. Bestenfalls eine intakte Version des mutierten Gens ließ sich ins Erbgut einschleusen. Erst heute berichten Forscher der kalifornischen Biotech-Firma Sangamo im Fachmagazin "Nature" von der ersten erfolgreichen Reparatur eines menschlichen Gens mit Hilfe eines neu entwickelten molekularen Werkzeugs. Und weisen der Gentherapie damit einen Ausweg aus der Krise, in der sie nach diversen Fehlschlägen und sogar tragischen Todesfällen steckt.
Es ist sicherlich kein Zufall, dass das Team um Michael Holmes für den Test des neuen Werkzeugs ausgerechnet die Erbkrankheit wählte, mit der die Euphorie um die Gentherapie begann: Am 14. September 1990 präsentierten US-Forscher die damals vierjährige Ashanti da Silva, deren Immunsystem durch ein mutiertes Gen lahm gelegt war.
SCID (Severe Combined Immunodeficiency) heißt die Krankheit, die sonst harmlose Keime zur tödlichen Bedrohung für Patienten macht. Die Forscher entnahmen dem Mädchen blutbildende Zellen und schleusten intakte Kopien des Gens ein. Und tatsächlich wuchsen genügend Immunzellen heran. Ashanti, vorher hermetisch von der Außenwelt abgeschirmt, konnte fortan ohne Angst vor Infektionen auf den Spielplatz.
Noch kein glücklicher Patient
Einen glücklichen Patienten kann Michael Holmes, Leiter der Studie und Forschungsdirektor bei Sangamo, leider noch nicht präsentieren. Er hat die neue Methode bisher nur an menschlichen Zellen in der Petrischale getestet. Die Wissenschaftler sind vorsichtiger geworden, seit die erste Euphorie verflogen ist und die ersten technischen Probleme aufgetreten sind. Unvergessen ist der Fall des 18-jährigen Jesse Gelsinger, der bei einem Gentherapie-Versuch starb. Unvergessen sind auch die drei SCID-kranken Kinder, die in Paris zunächst erfolgreich mit einer Gentherapie behandelt wurden, später aber an Blutkrebs erkrankten - ein Kind starb inzwischen.
Denn das ist die größte Gefahr beim Einschleusen von künstlicher DNA: Nachgebaute Gene können offenbar Krebsgene aktivieren, weil sie zufällig ins Erbgut eingebaut werden und dabei wichtige Erbinformationen zerstören.
Diese Gefahr lässt sich mit dem neuen Werkzeug vermeiden, da es aus zwei Komponenten besteht. Der so genannte Zinkfinger ist ein Protein, das eine bestimmte Abfolge der DNA-Bausteine erkennt. So lässt sich jede gewünschte Stelle im Erbgut ansteuern. Dann wird die zweite Komponente aktiv, eine Art molekulare Schere, die DNA schneidet. Durch den Schnitt wird ein natürlicher Reparaturmechanismus ausgelöst, bei dem die losen DNA-Enden nicht nur zusammengeführt, sondern auch die DNA-Bausteine vor und hinter dem Schnitt ersetzt werden. Damit die richtige und nicht wieder die mutierte Abfolge von DNA-Bausteinen ergänzt wird, stellen die Wissenschaftler außer ihrem Werkzeug auch noch ein Stück DNA mit der korrekten Gensequenz als Korrekturvorlage zur Verfügung.
Der "Quantensprung" der Sangamo-Forscher
Einen "Quantensprung" nennt Albert Jeltsch von der International University Bremen die Arbeit der Sangamo-Forscher neidlos. Der Molekularbiologe bastelt ähnliche Werkzeuge, indem er an den DNA-bindenden Zinkfinger eine Komponente hängt, die die DNA nicht schneidet, sondern nur verändert. Jeltsch hofft, Gene des Herpes-Virus, das beim Menschen Herpesbläschen verursacht, stillzulegen.
Sangamo will die Zinkfinger-Technologie vor allem zur Therapie von Erbkrankheiten einsetzen, die Blutzellen betreffen, wie zum Beispiel Sichelzellenanämie. Außerdem entwickelt das Unternehmen nach eigenen Angaben eine Therapie gegen HIV-Infektion. Dabei soll das Werkzeug in den Blutzellen ein Gen zerstören, das die Aids-Viren für die Infektion der T-Zellen benötigen.
Aaron Klug, Nobelpreisträger und Entdecker der Zinkfinger-Proteine aus dem britischen Cambridge, ist schon jetzt von den neuen Perspektiven begeistert: "Die Arbeit ist ein Meilenstein auf dem Weg zu genverändernden Therapien."
--------------------------------------------------------------------------------
Variantenreiche Wissenschaft
Herkömmlich Die intakte Version eines mutierten Gens wird ins Erbgut eingeschleust. Noch gibt es eine Reihe ungelöster technischer Probleme.
Neu Die Forscher lösen im Erbgut einen natürlichen Reparaturmechanismus aus. Dazu haben sie ein Werkzeug entwickelt, das sich aus einem speziellen Protein, dem so genannten Zinkfinger, und einer Art molekularen Schere zusammensetzt, mit der sich DNA schneiden lässt.
ftd.de, 03. 04. 2005
© 2005 Financial Times Deutschland, © Illustration: FTD/am; Quelle: Mark Benecke
Hier der Chart
Homepage
http://www.sangamo.com/news/news.php
Eine interessante Anlage
gruss meislo
Sangamo BioSciences Announces Publication in Nature Demonstrating the Use of Its Zinc Finger Technology to Correct Human Genes
Paper Describes Approach for `Genome Editing` That May Lead to Potential
Treatment for Monogenic Diseases and HIV/AIDS
RICHMOND, Calif., April 4 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) today announced publication of data that demonstrates the use of the Company`s zinc finger DNA-binding protein (ZFP) technology to achieve highly efficient, permanent correction of a disease-causing gene in primary human cells. This research, published in Nature as an advance online publication, represents a significant advance in the ability to specifically and efficiently modify the human genome and provides the scientific foundation for potential therapeutic approaches for a variety of genetic disorders and infectious diseases.
In this study, Sangamo scientists demonstrated the use of engineered zinc finger nucleases (ZFN(TM)) to correct errors in the DNA sequence of a disease-causing gene, the IL2Rgamma gene. Correction was achieved in a high percentage of treated cells without the need for selection. Importantly, gene correction was permanent and eliminated the need for integration of any foreign DNA sequence, a cause of problems in certain gene therapy studies.
"Using our ZFN technology we have advanced the field of targeted homologous recombination to levels of efficiency and specificity that could make potential therapeutic applications feasible," stated Michael C. Holmes, Ph.D., Director, Therapeutic Gene Modification at Sangamo and the study`s senior author. "Our ZFN technology allows us to facilitate modification of a DNA sequence at a very specific point in the genome, in this case, at the site of a mutation in a gene. The cell`s own machinery corrects the mutation using a DNA sequence that we provide. All of this happens without the need for integration of foreign DNA into the genome of cells. Once the gene is repaired, these cells undergo normal division and replication, resulting in daughter cells that carry the modified gene and thus are permanently corrected."
"For years, scientists have been searching for a way to modify or edit the genome of plants and animals in a precise and predictable fashion," said Nobel Laureate, Professor Sir Aaron Klug, of the MRC Laboratory of Molecular Biology, Cambridge, UK. "This work is therefore truly a landmark study that provides the foundation for gene modification-based therapeutics without the safety issues that have plagued many traditional gene therapy applications. It gives me great personal satisfaction to see this remarkable outcome of my original discovery of zinc fingers and their development."
Study Results
Mutations in the gene encoding the IL2Rgamma protein invariably cause X-linked SCID (X-linked Severe Combined Immunodeficiency Disease) or so-called Bubble-boy disease. Patients with such mutations do not produce a functional IL2Rgamma protein; never develop a functional immune system and die of severe infections within 12-18 months of birth.
In this study, highly specific engineered ZFNs designed to bind to sequences close to an X-linked SCID-causing mutation in the IL2Rgamma gene resulted in a high percentage of the cells undergoing gene correction. In addition, it was observed that approximately one third of the corrected cells acquired the desired modification on both chromosomes. The expected downstream changes in both RNA and protein levels were also observed. Comparably high levels of correction were observed in primary human T-cells. While further work will be required to optimize the system for therapeutic use, the gene correction efficiencies established here may be sufficient to achieve a therapeutic effect.
"I would like to congratulate all of the Sangamo scientists involved in the generation of these data," stated Edward Lanphier, Sangamo`s president and CEO. "It is gratifying that their work has been recognized by the prestigious journal Nature. These results highlight the potential for gene correction therapy for human monogenic disorders i.e. those diseases caused by mutation of a single gene. We are now working with our clinical collaborators to move this technology into the clinic. Our initial research will focus on monogenic diseases of blood cells such as Sickle Cell Anemia and beta-Thalassemia. In addition, the technology also forms the basis of our program to develop a potential therapeutic for HIV infection by disrupting expression of the CCR5 gene to generate a population of HIV-resistant cells."
About Zinc Finger DNA Binding Proteins
Zinc Finger DNA-binding Proteins (ZFPs) are a naturally occurring class of DNA binding proteins. The DNA recognition and binding function of ZFPs can be engineered and thus directed to a targeted sequence of DNA. This permits the delivery of a variety of functional domains to a gene-specific location. ZFPs are being developed for two significant therapeutic applications: gene regulation and gene modification. In the case of therapeutic gene regulation, ZFPs are being engineered to either turn on therapeutically beneficial genes or turn off the expression of disease-causing genes. For gene modification, ZFPs are being used in combination with a DNA cutting enzyme (endonuclease) functional domain to generate ZFNs that facilitate the correction of mutant gene sequences that cause disease or the disruption of genes that facilitate disease progression.
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development programs are currently in Phase I clinical trials for evaluation of safety in patients with peripheral artery disease and diabetic neuropathy. Other therapeutic development programs are focused on ischemic heart disease, congestive heart failure, cancer, neuropathic pain, and infectious and monogenic diseases. 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 TF(TM)) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for therapeutic gene modification as a treatment and possible cure for a variety of monogenic diseases such as sickle cell anemia and for infectious diseases such as HIV. For more information about Sangamo, visit the company`s web site at www.sangamo.com or www.expressinglife.com.
This press release may contain forward-looking statements based on Sangamo`s current expectations. These forward-looking statements include, without limitation, references to the research and development of novel ZFP TFs and ZFNs, clinical trials 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 technological challenges, Sangamo`s ability to develop commercially viable products and technological developments by our competitors. See the company`s SEC filings, and in particular, the risk factors described in the company`s Annual Report on Form 10-K and its most recent 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.
SOURCE Sangamo BioSciences, Inc.
-0- 04/04/2005
/CONTACT: Elizabeth Wolffe, Ph.D., of Sangamo BioSciences, Inc.,
+1-510-970-6000, ext. 271, or ewolffe@sangamo.com; or media, Kathy Nugent,
+1-212-213-0006, or investors, John Cummings, +1-415-352-6262, both of Burns
McClellan, Inc., for Sangamo BioSciences, Inc./
/Web site: http://www.sangamo.com /
(SGMO)
CO: Sangamo BioSciences, Inc.
ST: California
IN: MTC BIO HEA MAG PUB
SU: SVY
MP-MW
-- SFM053 --
1340 04/04/2005 07:00 EDT http://www.prnewswire.
http://biz.yahoo.com/prnews/050404/sfm053.html?.v=5
Paper Describes Approach for `Genome Editing` That May Lead to Potential
Treatment for Monogenic Diseases and HIV/AIDS
RICHMOND, Calif., April 4 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) today announced publication of data that demonstrates the use of the Company`s zinc finger DNA-binding protein (ZFP) technology to achieve highly efficient, permanent correction of a disease-causing gene in primary human cells. This research, published in Nature as an advance online publication, represents a significant advance in the ability to specifically and efficiently modify the human genome and provides the scientific foundation for potential therapeutic approaches for a variety of genetic disorders and infectious diseases.
In this study, Sangamo scientists demonstrated the use of engineered zinc finger nucleases (ZFN(TM)) to correct errors in the DNA sequence of a disease-causing gene, the IL2Rgamma gene. Correction was achieved in a high percentage of treated cells without the need for selection. Importantly, gene correction was permanent and eliminated the need for integration of any foreign DNA sequence, a cause of problems in certain gene therapy studies.
"Using our ZFN technology we have advanced the field of targeted homologous recombination to levels of efficiency and specificity that could make potential therapeutic applications feasible," stated Michael C. Holmes, Ph.D., Director, Therapeutic Gene Modification at Sangamo and the study`s senior author. "Our ZFN technology allows us to facilitate modification of a DNA sequence at a very specific point in the genome, in this case, at the site of a mutation in a gene. The cell`s own machinery corrects the mutation using a DNA sequence that we provide. All of this happens without the need for integration of foreign DNA into the genome of cells. Once the gene is repaired, these cells undergo normal division and replication, resulting in daughter cells that carry the modified gene and thus are permanently corrected."
"For years, scientists have been searching for a way to modify or edit the genome of plants and animals in a precise and predictable fashion," said Nobel Laureate, Professor Sir Aaron Klug, of the MRC Laboratory of Molecular Biology, Cambridge, UK. "This work is therefore truly a landmark study that provides the foundation for gene modification-based therapeutics without the safety issues that have plagued many traditional gene therapy applications. It gives me great personal satisfaction to see this remarkable outcome of my original discovery of zinc fingers and their development."
Study Results
Mutations in the gene encoding the IL2Rgamma protein invariably cause X-linked SCID (X-linked Severe Combined Immunodeficiency Disease) or so-called Bubble-boy disease. Patients with such mutations do not produce a functional IL2Rgamma protein; never develop a functional immune system and die of severe infections within 12-18 months of birth.
In this study, highly specific engineered ZFNs designed to bind to sequences close to an X-linked SCID-causing mutation in the IL2Rgamma gene resulted in a high percentage of the cells undergoing gene correction. In addition, it was observed that approximately one third of the corrected cells acquired the desired modification on both chromosomes. The expected downstream changes in both RNA and protein levels were also observed. Comparably high levels of correction were observed in primary human T-cells. While further work will be required to optimize the system for therapeutic use, the gene correction efficiencies established here may be sufficient to achieve a therapeutic effect.
"I would like to congratulate all of the Sangamo scientists involved in the generation of these data," stated Edward Lanphier, Sangamo`s president and CEO. "It is gratifying that their work has been recognized by the prestigious journal Nature. These results highlight the potential for gene correction therapy for human monogenic disorders i.e. those diseases caused by mutation of a single gene. We are now working with our clinical collaborators to move this technology into the clinic. Our initial research will focus on monogenic diseases of blood cells such as Sickle Cell Anemia and beta-Thalassemia. In addition, the technology also forms the basis of our program to develop a potential therapeutic for HIV infection by disrupting expression of the CCR5 gene to generate a population of HIV-resistant cells."
About Zinc Finger DNA Binding Proteins
Zinc Finger DNA-binding Proteins (ZFPs) are a naturally occurring class of DNA binding proteins. The DNA recognition and binding function of ZFPs can be engineered and thus directed to a targeted sequence of DNA. This permits the delivery of a variety of functional domains to a gene-specific location. ZFPs are being developed for two significant therapeutic applications: gene regulation and gene modification. In the case of therapeutic gene regulation, ZFPs are being engineered to either turn on therapeutically beneficial genes or turn off the expression of disease-causing genes. For gene modification, ZFPs are being used in combination with a DNA cutting enzyme (endonuclease) functional domain to generate ZFNs that facilitate the correction of mutant gene sequences that cause disease or the disruption of genes that facilitate disease progression.
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development programs are currently in Phase I clinical trials for evaluation of safety in patients with peripheral artery disease and diabetic neuropathy. Other therapeutic development programs are focused on ischemic heart disease, congestive heart failure, cancer, neuropathic pain, and infectious and monogenic diseases. 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 TF(TM)) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for therapeutic gene modification as a treatment and possible cure for a variety of monogenic diseases such as sickle cell anemia and for infectious diseases such as HIV. For more information about Sangamo, visit the company`s web site at www.sangamo.com or www.expressinglife.com.
This press release may contain forward-looking statements based on Sangamo`s current expectations. These forward-looking statements include, without limitation, references to the research and development of novel ZFP TFs and ZFNs, clinical trials 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 technological challenges, Sangamo`s ability to develop commercially viable products and technological developments by our competitors. See the company`s SEC filings, and in particular, the risk factors described in the company`s Annual Report on Form 10-K and its most recent 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.
SOURCE Sangamo BioSciences, Inc.
-0- 04/04/2005
/CONTACT: Elizabeth Wolffe, Ph.D., of Sangamo BioSciences, Inc.,
+1-510-970-6000, ext. 271, or ewolffe@sangamo.com; or media, Kathy Nugent,
+1-212-213-0006, or investors, John Cummings, +1-415-352-6262, both of Burns
McClellan, Inc., for Sangamo BioSciences, Inc./
/Web site: http://www.sangamo.com /
(SGMO)
CO: Sangamo BioSciences, Inc.
ST: California
IN: MTC BIO HEA MAG PUB
SU: SVY
MP-MW
-- SFM053 --
1340 04/04/2005 07:00 EDT http://www.prnewswire.
http://biz.yahoo.com/prnews/050404/sfm053.html?.v=5
Hier der Jahresabschlussbericht mit allen wichtigen informationen.
http://media.corporate-ir.net/media_files/irol/12/120938/rep…
gruss meislo
http://media.corporate-ir.net/media_files/irol/12/120938/rep…
gruss meislo
Sangamo BioSciences Announces Presentation of ZFN(TM) Gene Correction Data at American Society for Gene Therapy Stakeholders` Meeting
Thursday April 7, 7:00 am ET
RICHMOND, Calif., April 7 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO - News) today announced that Philip Gregory, D. Phil., Sangamo`s senior director of research will make a presentation at a Stakeholders` Meeting organized by the American Society of Gene Therapy. The meeting is being held in Arlington, Virginia, April 7-8, 2005. Dr. Gregory will discuss data that demonstrates the use of Sangamo`s zinc finger DNA-binding protein (ZFP) technology in a fundamentally new approach to therapeutics.
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The purpose of the meeting is for stakeholders in gene therapy (biotechnology, pharmaceutical companies, academic investigators, the Food and Drug Administration and the National Institutes of Health) to come together to assess progress in clinical gene transfer research and to discuss collaborative ways in which the field can be effectively advanced. ( http://www.asgt.org/stakeholders/overview.shtml )
"We are very pleased to contribute to this important meeting that is being held to assess the progress and potential of gene therapy," said Edward Lanphier, Sangamo`s president and CEO. "We believe our technology provides an alternative that may allow us to realize the potential of genetic approaches to disease while avoiding some of the pitfalls encountered in the past. Traditional gene addition approaches require insertion of a therapeutic gene under the control of a foreign promoter into the genome. In contrast, our technology does not require integration yet provides permanent correction of genes in cells at frequencies that we believe will be therapeutically relevant. "
The data that Dr. Gregory will present is described in an article that was published in Nature magazine as an advance online publication on April 3, 2005 ( http://www.nature.com/cgi- taf/dynapage.taf?file=/nature/journal/vaop/ncurrent/index.html ) and provides the scientific foundation for specific and efficient permanent modification of the human genome without the need for integration of foreign DNA sequences. The research addresses the modification of a common site for mutations in the gene encoding the IL2Rgamma protein that cause X-linked SCID (X-linked Severe Combined Immunodeficiency Disease) or so-called Bubble-boy disease. Patients with such mutations do not produce a functional IL2Rgamma protein, never develop a functional immune system and die of severe infections within 12-18 months of birth.
In the study, highly specific engineered ZFP nucleases (ZFN(TM)) designed to bind to sequences close to an X-linked SCID-causing mutation in the IL2Rgamma gene resulted in a high percentage of the cells undergoing gene correction. While further work will be required to optimize the system for therapeutic use, the gene correction efficiencies established here may be sufficient to achieve a therapeutic effect.
Sangamo scientists have engineered ZFNs that can be used to facilitate therapeutic gene correction or disruption. Sangamo is developing ZFNs for gene correction therapy for human monogenic disorders, i.e., those diseases caused by mutation of a single gene. The initial focus is on monogenic diseases of blood cells such as sickle cell anemia and beta-thalassemia. In addition, the technology also forms the basis of Sangamo`s program to develop a potential therapeutic for HIV infection by disrupting expression of the CCR5 gene in T-cells to make the cells resistant to HIV infection in order to provide HIV-infected individuals with a reservoir of healthy and uninfectable T-cells. CCR5 is a well-studied cell surface receptor that serves as a co-receptor for HIV entry into cells and is a well-validated target for HIV treatment, in part because individuals with a natural disruption of their CCR5 gene have been shown to be resistant to HIV infection.
About Sangamo
Sangamo BioSciences, Inc is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development programs are currently in Phase I clinical trials for evaluation of safety in patients with peripheral artery disease and diabetic neuropathy. Other therapeutic development programs are focused on ischemic heart disease, congestive heart failure, cancer, neuropathic pain, and infectious and monogenic diseases. 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 TF(TM)) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for therapeutic gene modification as a treatment and possible cure for a variety of monogenic diseases such as sickle cell anemia and for infectious diseases such as HIV. For more information about Sangamo, visit the company`s web site at www.sangamo.com or www.expressinglife.com.
Thursday April 7, 7:00 am ET
RICHMOND, Calif., April 7 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO - News) today announced that Philip Gregory, D. Phil., Sangamo`s senior director of research will make a presentation at a Stakeholders` Meeting organized by the American Society of Gene Therapy. The meeting is being held in Arlington, Virginia, April 7-8, 2005. Dr. Gregory will discuss data that demonstrates the use of Sangamo`s zinc finger DNA-binding protein (ZFP) technology in a fundamentally new approach to therapeutics.
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The purpose of the meeting is for stakeholders in gene therapy (biotechnology, pharmaceutical companies, academic investigators, the Food and Drug Administration and the National Institutes of Health) to come together to assess progress in clinical gene transfer research and to discuss collaborative ways in which the field can be effectively advanced. ( http://www.asgt.org/stakeholders/overview.shtml )
"We are very pleased to contribute to this important meeting that is being held to assess the progress and potential of gene therapy," said Edward Lanphier, Sangamo`s president and CEO. "We believe our technology provides an alternative that may allow us to realize the potential of genetic approaches to disease while avoiding some of the pitfalls encountered in the past. Traditional gene addition approaches require insertion of a therapeutic gene under the control of a foreign promoter into the genome. In contrast, our technology does not require integration yet provides permanent correction of genes in cells at frequencies that we believe will be therapeutically relevant. "
The data that Dr. Gregory will present is described in an article that was published in Nature magazine as an advance online publication on April 3, 2005 ( http://www.nature.com/cgi- taf/dynapage.taf?file=/nature/journal/vaop/ncurrent/index.html ) and provides the scientific foundation for specific and efficient permanent modification of the human genome without the need for integration of foreign DNA sequences. The research addresses the modification of a common site for mutations in the gene encoding the IL2Rgamma protein that cause X-linked SCID (X-linked Severe Combined Immunodeficiency Disease) or so-called Bubble-boy disease. Patients with such mutations do not produce a functional IL2Rgamma protein, never develop a functional immune system and die of severe infections within 12-18 months of birth.
In the study, highly specific engineered ZFP nucleases (ZFN(TM)) designed to bind to sequences close to an X-linked SCID-causing mutation in the IL2Rgamma gene resulted in a high percentage of the cells undergoing gene correction. While further work will be required to optimize the system for therapeutic use, the gene correction efficiencies established here may be sufficient to achieve a therapeutic effect.
Sangamo scientists have engineered ZFNs that can be used to facilitate therapeutic gene correction or disruption. Sangamo is developing ZFNs for gene correction therapy for human monogenic disorders, i.e., those diseases caused by mutation of a single gene. The initial focus is on monogenic diseases of blood cells such as sickle cell anemia and beta-thalassemia. In addition, the technology also forms the basis of Sangamo`s program to develop a potential therapeutic for HIV infection by disrupting expression of the CCR5 gene in T-cells to make the cells resistant to HIV infection in order to provide HIV-infected individuals with a reservoir of healthy and uninfectable T-cells. CCR5 is a well-studied cell surface receptor that serves as a co-receptor for HIV entry into cells and is a well-validated target for HIV treatment, in part because individuals with a natural disruption of their CCR5 gene have been shown to be resistant to HIV infection.
About Sangamo
Sangamo BioSciences, Inc is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development programs are currently in Phase I clinical trials for evaluation of safety in patients with peripheral artery disease and diabetic neuropathy. Other therapeutic development programs are focused on ischemic heart disease, congestive heart failure, cancer, neuropathic pain, and infectious and monogenic diseases. 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 TF(TM)) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for therapeutic gene modification as a treatment and possible cure for a variety of monogenic diseases such as sickle cell anemia and for infectious diseases such as HIV. For more information about Sangamo, visit the company`s web site at www.sangamo.com or www.expressinglife.com.
4,08 Dollar
Die 4-dollarmarke könnte heute geknackt werden!
gruss meislo
Die 4-dollarmarke könnte heute geknackt werden!
gruss meislo
Trading Spotlight
NEW YORK, February 18 (newratings.com) - In a research note published yesterday, analysts at JMP Securities maintain their "market outperform" rating on Sangamo BioSciences Inc (SGMO.NAS). The target price is set to $10.00.
gruss meislo
gruss meislo
sehr schön, keiner interessiert sich für die aktie, auch an der nasdaq sind die umsätze eher bescheiden, das sehe ich sehr positiv, die aktie scheint tatsächlich noch unentdeckt zu sein, dabei bietet die technologie faszinierende möglichkeiten, genau so stelle ich mir die medizin der zukunft vor: im gegensatz zur sonst so verbreiteten symptomdoktorei (die oftmals nur einen - längerfristig gesehen - nicht allzu aussichtsreichen versuch darstellt, das meist nicht ausreichend verstandene diffizile physiologische gleichgewicht des menschlichen organismus oberflächlich wiederherzustellen während die symptome dann nur zeitverzögert an gleicher oder anderer stelle und machmal auch verstärkt wieder hervortreten oder der organismus dafür in ganz anderen bereichen geschädigt wird (z.b. bei immunsuppressiva), wird hier endlich versucht an der ursache anzugreifen und das - im gegensatz zu viralen fähren z.b. - sehr behutsam und gezielt! ist die technlogie erstmal ausgereift, bieten sich hier natürlich grenzenlose möglichkeiten, die technologie könnte zur standardplattform werden, mit der viele krankheiten neu angegangen werden können!
ich denke, jetzt ist man noch recht früh dabei, gewinne werden sich sicherlich nicht gleich morgen einstellen - obwohl, wenn man den kursverlauf des vergangenen jahres mal als seitwärtsbewegung betrachtet, wir uns am unteren ende dieser befinden und es auch gut mal wieder gegen 5$ laufen könnte...
denke, der zeitpunkt ist nicht der schlechteste sich jetzt hier längerfristig zu positionieren und mal abwarten, ob es nicht mal wieder ein zuverlässiger "indikator" war, dass es bei wo innerhalb von 4 jahren gerade mal einen thread mit einer handvoll postings zu sangamo gibt! lol ;-)
ich denke, jetzt ist man noch recht früh dabei, gewinne werden sich sicherlich nicht gleich morgen einstellen - obwohl, wenn man den kursverlauf des vergangenen jahres mal als seitwärtsbewegung betrachtet, wir uns am unteren ende dieser befinden und es auch gut mal wieder gegen 5$ laufen könnte...
denke, der zeitpunkt ist nicht der schlechteste sich jetzt hier längerfristig zu positionieren und mal abwarten, ob es nicht mal wieder ein zuverlässiger "indikator" war, dass es bei wo innerhalb von 4 jahren gerade mal einen thread mit einer handvoll postings zu sangamo gibt! lol ;-)
Sangamo BioSciences Initiates Clinical Trial for Novel Therapy for Diabetic Neuropathy
THURSDAY, MAY 05, 2005 7:00 AM
- PR Newswire
RICHMOND, Calif., May 5, 2005 /PRNewswire-FirstCall via COMTEX/ -- Sangamo BioSciences, Inc. (SGMO) today announced that the company has initiated a Phase I clinical trial of SB-509, a novel therapeutic designed to protect and stimulate the regeneration of peripheral nerve function in diabetics suffering from peripheral neuropathy. The multi-center study is designed to evaluate clinical safety of SB-509 in diabetics with mild to moderate diabetic peripheral sensory motor neuropathy in the legs.
"We are successfully translating our zinc finger DNA-binding protein (ZFP) technology into products that are now being tested in human clinical trials to address significant unmet medical needs," said Edward Lanphier, Sangamo`s president and CEO. "This is an important step on our path to realizing our goal of building the first new therapeutic product development platform for the 21st century."
The U.S. Food and Drug Administration cleared an Investigational New Drug (IND) filing for the single blind, placebo-controlled, dose-escalation trial in February 2005. The trial began with the screening and treatment of the first patient at the Diabetes and Glandular Disease Clinic in San Antonio, Texas. Other trial sites are expected to begin recruitment shortly and Sangamo expects to have up to a total of 4 sites participating in the study.
"We are very excited to be involved in testing this unique approach to what has so far been an inevitable problem for the majority of long-term diabetics," stated Mark S. Kipnes, M.D., a clinical investigator for Sangamo and Endocrinologist at the Diabetes and Glandular Disease Clinic. "Currently, apart from very strict glucose control which is unattainable for most patients; there is nothing available to treat or protect the damaged nerves directly, only products, such as analgesics, that treat the symptoms."
SB-509 is an injectable formulation of plasmid DNA that encodes a zinc finger DNA-binding protein transcription factor (ZFP TFTM), designed to upregulate the vascular endothelial growth factor A (VEGF-A) gene. VEGF-A has been demonstrated to have direct neurotrophic and neuroprotective properties. In preclinical animal efficacy studies in a diabetic rat model, SB-509 has proven effective in protecting motor and sensory nerve function from disease-induced nerve damage. These data will be presented at the 65th Annual Scientific Sessions of the American Diabetes Association held June 10-14, 2005 in San Diego, California.
"In previous studies, VEGF-A has been shown to be efficacious for maintenance of nerve function in this condition and we believe that our approach of activating the patient`s own VEGF-A gene directly may have important advantages over introducing a cloned gene or recombinant protein," said Dale Ando, M.D., Sangamo`s vice-president of therapeutic development and chief medical officer. "Although this is primarily a safety study, all of the patients participating will be given treatment in one leg and placebo in the other. The trial is designed as a `single blinded` study therefore neither the treating doctor nor the patient knows which leg received the treatment. Patient safety will be monitored throughout the study, and visits at one, two, three and six months will include neurological examination and electrophysiological testing. The ability to compare parameters in the treated and untreated leg and in addition compare to baseline measurements may allow us to gain some preliminary data on the therapy`s effectiveness in improving patients` neurological functions and electrophysiological parameters."
It is expected that approximately 12 patients will be treated in the trial. Subjects will receive injections in a distribution that targets the major peripheral nerves in the legs and feet. The first dose level will be injected in a distribution to treat nerves in the foot, the second will be distributed to include nerves in the outside of the lower leg and foot, the third for the whole lower leg and foot and the fourth for the major nerves in the whole leg from the thigh down. The trial is expected to take approximately 12 months to screen and enroll patients and 6 months for patient follow-up. Patients interested in participating in this trial may visit the Sangamo website at http://www.sangamo.com/human/human_phase1_trial.html for a list of study sites and their contact information.
About Diabetic Neuropathy
Diabetic peripheral sensory motor neuropathy is one of the most frequent complications of diabetes and affects an estimated 50% of diabetics who have lived with their diabetes for ten years or more. The American Diabetes Association estimates that there are currently approximately 18.3 million people with diabetes in the United States. According to the CDC, diabetes is becoming more common in the United States. From 1980 through 2002, the number of Americans with diabetes more than doubled. Symptoms include numbness, tingling sensations and pain particularly in the toes or feet. This is gradually replaced by loss of sensation and motor function as nerve damage progresses. Ulcers and sores may appear on numb areas of the foot because pressure or injury goes unnoticed. Despite adequate treatment, these areas of trauma frequently become infected and this infection may spread to the bone, necessitating amputation of the leg or foot. More than 60% of non-traumatic lower-limb amputations in the United States occur among people with diabetes. In the period from 2000 to 2001 this translated to approximately 82,000 amputations.
Source: http://www.prnewswire.com
THURSDAY, MAY 05, 2005 7:00 AM
- PR Newswire
RICHMOND, Calif., May 5, 2005 /PRNewswire-FirstCall via COMTEX/ -- Sangamo BioSciences, Inc. (SGMO) today announced that the company has initiated a Phase I clinical trial of SB-509, a novel therapeutic designed to protect and stimulate the regeneration of peripheral nerve function in diabetics suffering from peripheral neuropathy. The multi-center study is designed to evaluate clinical safety of SB-509 in diabetics with mild to moderate diabetic peripheral sensory motor neuropathy in the legs.
"We are successfully translating our zinc finger DNA-binding protein (ZFP) technology into products that are now being tested in human clinical trials to address significant unmet medical needs," said Edward Lanphier, Sangamo`s president and CEO. "This is an important step on our path to realizing our goal of building the first new therapeutic product development platform for the 21st century."
The U.S. Food and Drug Administration cleared an Investigational New Drug (IND) filing for the single blind, placebo-controlled, dose-escalation trial in February 2005. The trial began with the screening and treatment of the first patient at the Diabetes and Glandular Disease Clinic in San Antonio, Texas. Other trial sites are expected to begin recruitment shortly and Sangamo expects to have up to a total of 4 sites participating in the study.
"We are very excited to be involved in testing this unique approach to what has so far been an inevitable problem for the majority of long-term diabetics," stated Mark S. Kipnes, M.D., a clinical investigator for Sangamo and Endocrinologist at the Diabetes and Glandular Disease Clinic. "Currently, apart from very strict glucose control which is unattainable for most patients; there is nothing available to treat or protect the damaged nerves directly, only products, such as analgesics, that treat the symptoms."
SB-509 is an injectable formulation of plasmid DNA that encodes a zinc finger DNA-binding protein transcription factor (ZFP TFTM), designed to upregulate the vascular endothelial growth factor A (VEGF-A) gene. VEGF-A has been demonstrated to have direct neurotrophic and neuroprotective properties. In preclinical animal efficacy studies in a diabetic rat model, SB-509 has proven effective in protecting motor and sensory nerve function from disease-induced nerve damage. These data will be presented at the 65th Annual Scientific Sessions of the American Diabetes Association held June 10-14, 2005 in San Diego, California.
"In previous studies, VEGF-A has been shown to be efficacious for maintenance of nerve function in this condition and we believe that our approach of activating the patient`s own VEGF-A gene directly may have important advantages over introducing a cloned gene or recombinant protein," said Dale Ando, M.D., Sangamo`s vice-president of therapeutic development and chief medical officer. "Although this is primarily a safety study, all of the patients participating will be given treatment in one leg and placebo in the other. The trial is designed as a `single blinded` study therefore neither the treating doctor nor the patient knows which leg received the treatment. Patient safety will be monitored throughout the study, and visits at one, two, three and six months will include neurological examination and electrophysiological testing. The ability to compare parameters in the treated and untreated leg and in addition compare to baseline measurements may allow us to gain some preliminary data on the therapy`s effectiveness in improving patients` neurological functions and electrophysiological parameters."
It is expected that approximately 12 patients will be treated in the trial. Subjects will receive injections in a distribution that targets the major peripheral nerves in the legs and feet. The first dose level will be injected in a distribution to treat nerves in the foot, the second will be distributed to include nerves in the outside of the lower leg and foot, the third for the whole lower leg and foot and the fourth for the major nerves in the whole leg from the thigh down. The trial is expected to take approximately 12 months to screen and enroll patients and 6 months for patient follow-up. Patients interested in participating in this trial may visit the Sangamo website at http://www.sangamo.com/human/human_phase1_trial.html for a list of study sites and their contact information.
About Diabetic Neuropathy
Diabetic peripheral sensory motor neuropathy is one of the most frequent complications of diabetes and affects an estimated 50% of diabetics who have lived with their diabetes for ten years or more. The American Diabetes Association estimates that there are currently approximately 18.3 million people with diabetes in the United States. According to the CDC, diabetes is becoming more common in the United States. From 1980 through 2002, the number of Americans with diabetes more than doubled. Symptoms include numbness, tingling sensations and pain particularly in the toes or feet. This is gradually replaced by loss of sensation and motor function as nerve damage progresses. Ulcers and sores may appear on numb areas of the foot because pressure or injury goes unnoticed. Despite adequate treatment, these areas of trauma frequently become infected and this infection may spread to the bone, necessitating amputation of the leg or foot. More than 60% of non-traumatic lower-limb amputations in the United States occur among people with diabetes. In the period from 2000 to 2001 this translated to approximately 82,000 amputations.
Source: http://www.prnewswire.com
Die Präsidentin der Amerikanischen Gesellschaft für Gentherapie (ASGT) äussert sich sehr positiv zu jüngsten Forschungsergebnissen von Sangamo:
Sangamo BioSciences` Gene Correction Technology, Published in Nature Magazine, Is Seen as `Considerable Step Towards a Successful Genetic- Engineering Approach to Treating Human Disease`
THURSDAY, JUNE 02, 2005 7:00 AM
RICHMOND, Calif., June 2, 2005 /PRNewswire-FirstCall via COMTEX/ -- Sangamo BioSciences, Inc. (SGMO) today announced that Company research published in an article in the June 2 print issue of Nature magazine represents a significant advance in the ability to specifically and efficiently modify the human genome and provides the scientific foundation for potential therapeutic approaches for a variety of genetic disorders and infectious diseases. The research data, previously released in an advanced online publication, demonstrate the use of the Company`s zinc finger DNA-binding protein (ZFP) technology to achieve highly efficient, permanent correction of a disease-causing gene in primary human cells.
The June 2 issue of Nature also features a review of Sangamo`s work written by Katherine A. High, M.D., who characterized the work as
"a considerable step towards a successful genetic-engineering approach to treating human disease." Dr. High is a Howard Hughes Medical Institute Investigator, an attending physician in the Hematology Division of The Children`s Hospital of Philadelphia and the current President of the American Society of Gene Therapy (ASGT).
In this study, Sangamo scientists demonstrated the use of engineered zinc finger nucleases (ZFNTM) to correct errors in the DNA sequence of the IL2R-gamma gene that causes X-linked severe combined immunodeficiency disease (X-linked SCID). Correction was achieved in a high percentage of treated cells without the need for selection. Importantly, gene correction was site-specific, permanent and eliminated the need for integration of any foreign DNA sequence, a cause of problems in certain gene therapy studies.
"The recognition and overwhelmingly positive response to this work by the scientific and medical community is gratifying," said Dale Ando, M.D., Sangamo`s vice president of therapeutics and Chief Medical Officer. "As Dr. High pointed out in her News and Views article, most gene therapies are gene-addition therapies. However, the ultimate aim is to correct a mutation and thus enable the production of a normal protein under the control of the cell`s intrinsic regulatory signals. Until now this goal has remained out of reach for human therapeutics. The ZFN(TM) technology that we have developed provides a potential solution to specifically and permanently correcting a mistake in a cellular gene without the need for integration of foreign DNA into the genome."
In her review, Dr. High comments that Sangamo`s ZFN(TM) strategy for site-specific gene correction combines two biological processes that are highly evolutionarily conserved: 1) the targeting of a functional domain to a specific DNA sequence using a ZFP, in this case the functional domain is an enzyme that causes a break in the DNA strand, and 2) the repair of that break by a natural process known as homologous recombination. As she goes on to explain, "the most remarkable feature of this complex process is the frequency at which it occurs." The authors show rates of gene correction by homologous recombination in cultured cells that are 15,000 to 20,000 times higher when a break is introduced by ZFNs than when no break is introduced. Referring to Sangamo`s data on correction of a mutation responsible for X-linked SCID, Dr. High stated that more work will be necessary before the technique can successfully be translated into humans but points out that if the rates of correction that have been achieved by the authors in T-cells can be recapitulated in CD34-expressing hematopoietic stem cells, "then they will probably be adequate to restore immune function."
"I would once again like to acknowledge and congratulate my colleagues at Sangamo on this ground-breaking work," said Edward Lanphier, Sangamo`s president and CEO. "We look forward to presenting these findings and data from related ZFNTM programs in sickle cell anemia, Wiskott-Aldrich Syndrome and HIV/AIDS this week at the Annual ASGT meeting being held in St. Louis. Our goal is to move these programs efficiently through preclinical testing and to work with our clinical collaborators to advance this technology into the clinic to benefit patients."
Study Results
Mutations in the gene encoding the IL2R-gamma protein invariably cause X-linked SCID or so-called Bubble-boy disease. Patients with such mutations do not produce a functional IL2R-gamma protein; never develop a functional immune system and die of severe infections within 12-18 months of birth.
In Sangamo`s study, highly specific engineered ZFNs designed to bind to sequences close to an X-linked SCID-causing mutation in the IL2R-gamma gene resulted in a high percentage of the cells undergoing gene correction. In addition, it was observed that approximately one third of the corrected cells acquired the desired modification on both chromosomes. The expected downstream changes in both RNA and protein levels were also observed. Comparably high levels of correction were observed in primary human T-cells. While further work will be required to optimize the system for therapeutic use, the gene correction efficiencies established here may be sufficient to achieve a therapeutic effect.
About Zinc Finger DNA Binding Proteins
Zinc Finger DNA-binding Proteins (ZFPs) are a naturally occurring class of DNA binding proteins. The DNA recognition and binding function of ZFPs can be engineered and thus directed to a targeted sequence of DNA. This permits the delivery of a variety of functional domains to a gene-specific location. ZFPs are being developed for two significant therapeutic applications: gene regulation and gene modification. In the case of therapeutic gene regulation, ZFPs are being engineered to either turn on therapeutically beneficial genes or turn off the expression of disease-causing genes. For gene modification, ZFPs are being used in combination with a DNA cutting enzyme (endonuclease) functional domain to generate ZFNs that facilitate the correction of mutant gene sequences that cause disease or the disruption of genes that facilitate disease progression.
http://www.prnewswire.com
quelle: http://www.bigcharts.com
Sangamo BioSciences` Gene Correction Technology, Published in Nature Magazine, Is Seen as `Considerable Step Towards a Successful Genetic- Engineering Approach to Treating Human Disease`
THURSDAY, JUNE 02, 2005 7:00 AM
RICHMOND, Calif., June 2, 2005 /PRNewswire-FirstCall via COMTEX/ -- Sangamo BioSciences, Inc. (SGMO) today announced that Company research published in an article in the June 2 print issue of Nature magazine represents a significant advance in the ability to specifically and efficiently modify the human genome and provides the scientific foundation for potential therapeutic approaches for a variety of genetic disorders and infectious diseases. The research data, previously released in an advanced online publication, demonstrate the use of the Company`s zinc finger DNA-binding protein (ZFP) technology to achieve highly efficient, permanent correction of a disease-causing gene in primary human cells.
The June 2 issue of Nature also features a review of Sangamo`s work written by Katherine A. High, M.D., who characterized the work as
"a considerable step towards a successful genetic-engineering approach to treating human disease." Dr. High is a Howard Hughes Medical Institute Investigator, an attending physician in the Hematology Division of The Children`s Hospital of Philadelphia and the current President of the American Society of Gene Therapy (ASGT).
In this study, Sangamo scientists demonstrated the use of engineered zinc finger nucleases (ZFNTM) to correct errors in the DNA sequence of the IL2R-gamma gene that causes X-linked severe combined immunodeficiency disease (X-linked SCID). Correction was achieved in a high percentage of treated cells without the need for selection. Importantly, gene correction was site-specific, permanent and eliminated the need for integration of any foreign DNA sequence, a cause of problems in certain gene therapy studies.
"The recognition and overwhelmingly positive response to this work by the scientific and medical community is gratifying," said Dale Ando, M.D., Sangamo`s vice president of therapeutics and Chief Medical Officer. "As Dr. High pointed out in her News and Views article, most gene therapies are gene-addition therapies. However, the ultimate aim is to correct a mutation and thus enable the production of a normal protein under the control of the cell`s intrinsic regulatory signals. Until now this goal has remained out of reach for human therapeutics. The ZFN(TM) technology that we have developed provides a potential solution to specifically and permanently correcting a mistake in a cellular gene without the need for integration of foreign DNA into the genome."
In her review, Dr. High comments that Sangamo`s ZFN(TM) strategy for site-specific gene correction combines two biological processes that are highly evolutionarily conserved: 1) the targeting of a functional domain to a specific DNA sequence using a ZFP, in this case the functional domain is an enzyme that causes a break in the DNA strand, and 2) the repair of that break by a natural process known as homologous recombination. As she goes on to explain, "the most remarkable feature of this complex process is the frequency at which it occurs." The authors show rates of gene correction by homologous recombination in cultured cells that are 15,000 to 20,000 times higher when a break is introduced by ZFNs than when no break is introduced. Referring to Sangamo`s data on correction of a mutation responsible for X-linked SCID, Dr. High stated that more work will be necessary before the technique can successfully be translated into humans but points out that if the rates of correction that have been achieved by the authors in T-cells can be recapitulated in CD34-expressing hematopoietic stem cells, "then they will probably be adequate to restore immune function."
"I would once again like to acknowledge and congratulate my colleagues at Sangamo on this ground-breaking work," said Edward Lanphier, Sangamo`s president and CEO. "We look forward to presenting these findings and data from related ZFNTM programs in sickle cell anemia, Wiskott-Aldrich Syndrome and HIV/AIDS this week at the Annual ASGT meeting being held in St. Louis. Our goal is to move these programs efficiently through preclinical testing and to work with our clinical collaborators to advance this technology into the clinic to benefit patients."
Study Results
Mutations in the gene encoding the IL2R-gamma protein invariably cause X-linked SCID or so-called Bubble-boy disease. Patients with such mutations do not produce a functional IL2R-gamma protein; never develop a functional immune system and die of severe infections within 12-18 months of birth.
In Sangamo`s study, highly specific engineered ZFNs designed to bind to sequences close to an X-linked SCID-causing mutation in the IL2R-gamma gene resulted in a high percentage of the cells undergoing gene correction. In addition, it was observed that approximately one third of the corrected cells acquired the desired modification on both chromosomes. The expected downstream changes in both RNA and protein levels were also observed. Comparably high levels of correction were observed in primary human T-cells. While further work will be required to optimize the system for therapeutic use, the gene correction efficiencies established here may be sufficient to achieve a therapeutic effect.
About Zinc Finger DNA Binding Proteins
Zinc Finger DNA-binding Proteins (ZFPs) are a naturally occurring class of DNA binding proteins. The DNA recognition and binding function of ZFPs can be engineered and thus directed to a targeted sequence of DNA. This permits the delivery of a variety of functional domains to a gene-specific location. ZFPs are being developed for two significant therapeutic applications: gene regulation and gene modification. In the case of therapeutic gene regulation, ZFPs are being engineered to either turn on therapeutically beneficial genes or turn off the expression of disease-causing genes. For gene modification, ZFPs are being used in combination with a DNA cutting enzyme (endonuclease) functional domain to generate ZFNs that facilitate the correction of mutant gene sequences that cause disease or the disruption of genes that facilitate disease progression.
http://www.prnewswire.com
quelle: http://www.bigcharts.com
Sangamo BioSciences Highlights Data From the 8th Annual Meeting of the American Society of Gene Therapy
Tuesday June 7, 7:00 am ET
Data Presented from ZFP-mediated Gene Regulation and Gene Modification Programs
RICHMOND, Calif., June 7 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO - News) announced today highlights of data presented at the 8th Annual Meeting of the American Society of Gene Therapy (ASGT) held in St. Louis, June 1-5, 2005. Sangamo scientists and their collaborators gave a total of nine presentations related to the Company`s proprietary zinc finger technology.
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Edward Lanphier, Sangamo`s president and CEO, stated, "There was an enormous amount of interest in our ZFP Technology platform at ASGT, particularly given the simultaneous publication in Nature of an article describing our ZFN gene correction technology. Needless to say, all of us at Sangamo are very proud of the high caliber of this research and its enthusiastic reception and look forward to additional presentations at the upcoming annual meeting of the International Society for Stem Cell Research in San Francisco later this month."
ASGT Meeting Highlights - Gene Modification
Sangamo is developing engineered zinc finger nucleases (ZFNTM) to correct errors in the DNA sequence of disease-causing genes for so-called monogenic diseases or to disrupt genes that facilitate disease. In a poster presentation and a Scientific Symposium Session podium presentation by Dale Ando, M.D., Sangamo`s vice president of therapeutic development and chief medical officer, data were presented showing that engineered ZFNs could be used to successfully disrupt the expression of the gene for CCR5. CCR5 is a cell surface receptor that serves as a co-receptor for HIV entry into cells and is a well-validated target for HIV treatment. A naturally occurring mutation of the CCR5 gene is present in a small percentage of the population and has been shown to confer resistance to HIV infection. Disruption of the CCR5 gene in T-cells or hematopoietic stem cells using Sangamo`s ZFN Technology could provide patients with an HIV-resistant T-cell population, thus helping to fight infection or progression of the disease. The Company`s goal is to begin human testing of this novel therapeutic approach in 2006.
ASGT Meeting Highlights - Gene Regulation
Sangamo is applying its ZFP Technology for therapeutic gene regulation using engineered zinc finger protein transcription factors (ZFP TFTM) to either turn on therapeutically beneficial genes or turn off the expression of disease-causing genes. In a podium presentation, Steve Zhang, Ph.D., Team Leader of Therapeutic Gene Regulation, described data from Sangamo`s program to develop a ZFP Therapeutic for the treatment of congestive heart failure. When a ZFP TF designed to selectively and efficiently turn off the phospholamban (PLN) gene was delivered into the hearts of rats, improved calcium flux and contractility were seen in the heart muscle cells. Previous research has shown that repression of PLN, a critical regulator of cardiac homeostasis and muscle contractility, leads to improved cardiac contractile properties in animal models of congestive heart failure.
A second presentation demonstrated that Sangamo scientists have designed ZFP TFs that specifically repress the expression of the gene that codes for tyrosine kinase receptor A (TrkA) in isolated rat dorsal root ganglion (DRG) neurons as well as human cell lines. TrkA is a well-validated pain target, and DRG neurons are key in the propagation of the pain signal. The reported data support the Company`s ongoing pre-clinical evaluation of ZFP TFs in additional animal models of neuropathic pain.
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development programs are currently in Phase I clinical trials for evaluation of safety in patients with peripheral artery disease and diabetic neuropathy. Other therapeutic development programs are focused on ischemic heart disease, congestive heart failure, cancer, neuropathic pain, and infectious and monogenic diseases. 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 TFTM) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for therapeutic gene modification as a treatment and possible cure for a variety of monogenic diseases, such as sickle cell anemia, and for infectious diseases such as HIV. For more information about Sangamo, visit the company`s web site at www.sangamo.com or www.expressinglife.com
This press release may contain forward-looking statements based on Sangamo`s current expectations. These forward-looking statements include, without limitation, references to the research and development of novel ZFP TFs and ZFNs, clinical trials 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 technological challenges, Sangamo`s ability to develop commercially viable products and technological developments by our competitors. See the company`s SEC filings, and in particular, the risk factors described in the company`s Annual Report on Form 10-K and its most recent 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.
--------------------------------------------------------------------------------
Source: Sangamo BioSciences, Inc.
Tuesday June 7, 7:00 am ET
Data Presented from ZFP-mediated Gene Regulation and Gene Modification Programs
RICHMOND, Calif., June 7 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO - News) announced today highlights of data presented at the 8th Annual Meeting of the American Society of Gene Therapy (ASGT) held in St. Louis, June 1-5, 2005. Sangamo scientists and their collaborators gave a total of nine presentations related to the Company`s proprietary zinc finger technology.
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Edward Lanphier, Sangamo`s president and CEO, stated, "There was an enormous amount of interest in our ZFP Technology platform at ASGT, particularly given the simultaneous publication in Nature of an article describing our ZFN gene correction technology. Needless to say, all of us at Sangamo are very proud of the high caliber of this research and its enthusiastic reception and look forward to additional presentations at the upcoming annual meeting of the International Society for Stem Cell Research in San Francisco later this month."
ASGT Meeting Highlights - Gene Modification
Sangamo is developing engineered zinc finger nucleases (ZFNTM) to correct errors in the DNA sequence of disease-causing genes for so-called monogenic diseases or to disrupt genes that facilitate disease. In a poster presentation and a Scientific Symposium Session podium presentation by Dale Ando, M.D., Sangamo`s vice president of therapeutic development and chief medical officer, data were presented showing that engineered ZFNs could be used to successfully disrupt the expression of the gene for CCR5. CCR5 is a cell surface receptor that serves as a co-receptor for HIV entry into cells and is a well-validated target for HIV treatment. A naturally occurring mutation of the CCR5 gene is present in a small percentage of the population and has been shown to confer resistance to HIV infection. Disruption of the CCR5 gene in T-cells or hematopoietic stem cells using Sangamo`s ZFN Technology could provide patients with an HIV-resistant T-cell population, thus helping to fight infection or progression of the disease. The Company`s goal is to begin human testing of this novel therapeutic approach in 2006.
ASGT Meeting Highlights - Gene Regulation
Sangamo is applying its ZFP Technology for therapeutic gene regulation using engineered zinc finger protein transcription factors (ZFP TFTM) to either turn on therapeutically beneficial genes or turn off the expression of disease-causing genes. In a podium presentation, Steve Zhang, Ph.D., Team Leader of Therapeutic Gene Regulation, described data from Sangamo`s program to develop a ZFP Therapeutic for the treatment of congestive heart failure. When a ZFP TF designed to selectively and efficiently turn off the phospholamban (PLN) gene was delivered into the hearts of rats, improved calcium flux and contractility were seen in the heart muscle cells. Previous research has shown that repression of PLN, a critical regulator of cardiac homeostasis and muscle contractility, leads to improved cardiac contractile properties in animal models of congestive heart failure.
A second presentation demonstrated that Sangamo scientists have designed ZFP TFs that specifically repress the expression of the gene that codes for tyrosine kinase receptor A (TrkA) in isolated rat dorsal root ganglion (DRG) neurons as well as human cell lines. TrkA is a well-validated pain target, and DRG neurons are key in the propagation of the pain signal. The reported data support the Company`s ongoing pre-clinical evaluation of ZFP TFs in additional animal models of neuropathic pain.
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The most advanced ZFP Therapeutic(TM) development programs are currently in Phase I clinical trials for evaluation of safety in patients with peripheral artery disease and diabetic neuropathy. Other therapeutic development programs are focused on ischemic heart disease, congestive heart failure, cancer, neuropathic pain, and infectious and monogenic diseases. 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 TFTM) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for therapeutic gene modification as a treatment and possible cure for a variety of monogenic diseases, such as sickle cell anemia, and for infectious diseases such as HIV. For more information about Sangamo, visit the company`s web site at www.sangamo.com or www.expressinglife.com
This press release may contain forward-looking statements based on Sangamo`s current expectations. These forward-looking statements include, without limitation, references to the research and development of novel ZFP TFs and ZFNs, clinical trials 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 technological challenges, Sangamo`s ability to develop commercially viable products and technological developments by our competitors. See the company`s SEC filings, and in particular, the risk factors described in the company`s Annual Report on Form 10-K and its most recent 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.
--------------------------------------------------------------------------------
Source: Sangamo BioSciences, Inc.
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