Click here to close now.




















Welcome!

News Feed Item

Sangamo BioSciences Presents Clinical Data From HIV Study Demonstrating Sustained Control Of Viremia

Reduction of Viral Load at or Below Limit of Detection Ongoing at 14 Weeks

RICHMOND, Calif., Oct. 28, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today the presentation of new data demonstrating sustained control of HIV viral load (VL) at or below the limit of detection for 14 weeks (at last measurement) in an SB-728-T- treated  HIV-infected subject who was not on antiretroviral therapy (ART).  The CCR5 delta-32 heterozygote subject is enrolled in Sangamo's clinical trial (SB-728-902 Cohort 5) and, as part of the clinical trial protocol, is undergoing an ART treatment interruption (TI), which is ongoing.

(Logo: http://photos.prnewswire.com/prnh/20130102/SF35903LOGO)

Data were presented at the Annual Meeting of the European Society of Gene and Cell Therapy (ESGCT and SETGyC Collaborative Congress) which is being held in Madrid from October 25-28, 2013.

"These data demonstrate that sustained functional control of HIV in the absence of ART is possible with a single SB-728-T treatment," stated Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "Our aim is to provide a population of immune memory cells that are protected from HIV infection and are capable of generating an effective immune response against the virus throughout the body. These data represent a further step toward demonstrating the efficacy and durability of this therapeutic approach."

Dr. Nichol added, "We continue to follow these Cohort 5 subjects and look forward to presenting a complete data set from this study, and a second ongoing trial (SB-728-1101), designed to maximize the engraftment of SB-728-T in subjects who are not CCR5 delta-32 heterozygotes, later this year."

Data from Sangamo's Phase 1 and 2 studies demonstrate that VL became undetectable during a TI from ART in three of seven evaluable CCR5 delta-32 heterozygote HIV-infected subjects, including two of six subjects that had completed TI in the ongoing SB-728-902 Cohort 5 study and an additional CCR5 delta-32 heterozygote subject from an earlier Phase 1 clinical trial of SB-728-T.  In one SB-728-902 Cohort 5 subject, VL has remained undetectable (at or below the limits of quantification of the current ultra-sensitive assays for HIV) for 14 weeks (to last measurement taken) and the TI is ongoing.  Reduction in VL from peak during TI showed a statistically significant correlation (p=0.015) with estimated numbers of engrafted ZFN modified cells (SB-728-T) in which both copies of the CCR5 gene had been disrupted (biallelic modification), in line with previously presented data from this program.

Collectively, data from these studies demonstrate that in all trial subjects, SB-728-T treatment results in a durable increase in total CD4 T-cells.  In addition, seven of nine subjects enrolled in Sangamo's Phase 1 study (SB-728-902 Cohorts1-3) experienced a longer term reduction in the viral reservoir as measured by HIV DNA in peripheral blood mononuclear cells, a source of chronic HIV infection not addressed by current ART.

Sangamo scientists and collaborators also presented data from preclinical and research programs in hemophilia and lysosomal storage disorders, hemoglobinopathies, cancer, cystic fibrosis, immunodeficiencies and the application of ZFN-mediated CCR5 modification in stem cells for HIV.

"The presentations at the ESGCT meeting demonstrate the diversity and breadth of potential therapeutic applications of Sangamo's ZFP technology," said Edward Lanphier, Sangamo's president and CEO. "We look forward to continuing to update on our progress in the coming months at a translational medicine meeting organized by The Lancet entitled, 'What Will it Take to Achieve an AIDS-free World?' in San Francisco, from November 3-5, as well as at the Sixth International Workshop on HIV Persistence, Reservoirs & Eradication Strategies in Miami in early December.  In addition, we will present data from Sangamo's preclinical programs at the Annual Meetings of the Society for Neuroscience in November and the American Society of Hematology (ASH) in early December."

Presentations at the ESGCT Meeting
Saturday, October 26

  • "TCR gene editing for the treatment of hematological malignancies." Chiara Bonini, San Raffaele Telethon Institute for Gene Therapy, Milan
  • "Efficient site-specific integration and in situ gene correction of human long-term repopulating hematopoietic stem cells by zinc finger nucleases." Pietro Genovese, San Raffaele Telethon Institute for Gene Therapy, Milan
  • "Allele-preferred targeted correction of CFTR gene in Cystic Fibrosis induced pluripotent stem cells." Brian Davis, University of Texas

Sunday, October 27

  • "Gene therapy for sickle cell disease." Donald Kohn, University of California, Los Angeles
  • "Genome editing with zinc finger nucleases." Michael Holmes, Sangamo BioSciences, Inc.
  • "Targeted transgene integration in human hematopoietic stem cells and induced pluripotent stem cells from normal donors and SCID-X1 patients." Angelo Lombardo, San Raffaele Telethon Institute for Gene Therapy, Milan
  • "Zinc finger nucleases targeting the beta-globin locus drive efficient correction of the sickle mutation in CD34+ cells." Megan Hoban, University of California, Los Angeles
  • "Rescue of T-cell deficiency in Prkdc mice by transplantation of gene-edited hematopoietic cells." Rafael Yanez, Royal Holloway, University of London
  • "ZFN mediated targeting of albumin: a platform for expression of multiple therapeutic genes in vivo." Xavier Anguela, The Children's Hospital of Philadelphia
  • "Development of ZFN-based preclinical in vitro cell model in human embryonic stem cells for Wiskott-Aldrich syndrome." Pilar Munoz-Fernandez, GENYO, Pfizer-University of Granada.

Monday, October 28

  • "Infusion of ZFN CCR5-modified CD4 T-cells (SB-728-T) led to long term reconstitution of CD4 T-cells and reduction of HIV-DNA levels in HIV-infected subjects on ART." Dale Ando, Sangamo BioSciences, Inc.

Summary of Clinical Trial Design
About SB-728-902 Cohort 5
Ten HIV-infected subjects heterozygous for the CCR5 delta-32 mutation (i.e. with one CCR5 gene that is naturally modified) who are currently on ART have been enrolled and have received a single intravenous infusion of SB-728-T (5 to 30 billion modified cells). Two months after SB-728-T treatment, subjects undergo a 16 week TI during which time their ART is discontinued. ART is reinstituted in subjects whose CD4 T-cell counts drop to <350 cells/ mm3 and/or whose HIV-RNA increases to >100,000 /mL for three consecutive weekly measurements. At the end of the TI, subjects with a sustained detectable HIV viral load are reinstituted on ART. Subjects with an undetectable viral load can remain off ART until HIV RNA levels are detectable or their CD4 T-cell count drops below 350 cell/mm3 for three consecutive weekly measurements.

A total of ten subjects have been treated in this cohort. 

Of the six evaluable subjects, we observed two subjects in which their VL became undetectable during TI from ART:

  • In one subject, VL suppression at, or below, the limit of quantification (LOQ) of virus was sustained from week 11 – 25 of TI and the TI is ongoing.
  • In the second subject there was a transient suppression of VL at or below LOQ.
  • A third subject completed the TI with 1-log decrease in VL from peak.

In three subjects, there was no reduction in VL during the TI, one completed the TI and in two the TI was terminated early due to their viral loads exceeding the upper limit allowed in the protocol. A seventh subject has not completed TI and is still being evaluated.

About SB-728-902 Cohorts 1-3
The study is an open-label Phase 1 clinical trial to evaluate the safety and tolerability of single infusions of an escalating dose of an autologous (a patient's own) CD4+ T-cell product genetically modified at the CCR5 gene by CCR5-specific ZFNs (SB-728-T). The trial enrolled nine HIV-infected subjects (three cohorts of three subjects each) who have sub-optimal T-cell levels and no detectable viral load on long-term ART.  Subjects remained on their existing antiviral therapy while receiving treatment with SB-728-T.

About SB-728-T
Sangamo's drug, SB-728-T, is generated by ZFN-mediated modification of the gene encoding the CCR5 receptor in a patient's own T-cells.  ZFN modification disrupts the expression of this key co-receptor for HIV entry and renders cells resistant to HIV infection. The approach is based on the observation that a naturally occurring mutation in the CCR5 gene, CCR5 delta-32, provides protection from HIV infection. Individuals in whom both copies of the CCR5 gene carry the delta-32 mutation are generally not susceptible to the most common strain of HIV.

About Sangamo
Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. The Company has ongoing Phase 2 and Phase1/2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic® for the treatment of HIV/AIDS. As part of its acquisition of Ceregene Inc., Sangamo acquired a fully-enrolled and funded, double-blind, placebo-controlled Phase 2 trial to evaluate NGF-AAV (CERE-110) in Alzheimer's disease. Sangamo's other therapeutic programs are focused on monogenic diseases, including hemophilia, Huntington's disease and  hemoglobinopathies such as beta-thalassemia and sickle cell anemia. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs).  Engineering of ZFPs that recognize a specific DNA sequence enables the creation of sequence-specific ZFP Nucleases (ZFNs) for gene modification and ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo has entered into a strategic collaboration with Shire AG to develop therapeutics for hemophilia, Huntington's disease and other monogenic diseases and 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, 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 for HIV/AIDS, the ability of a ZFP Therapeutic to control HIV infection, projected timing of release of SB-728-T clinical data, 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 public filings with the Securities and Exchange Commission, 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.

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
SYS-CON Events announced today that MobiDev, a software development company, will exhibit at the 17th International Cloud Expo®, which will take place November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. MobiDev is a software development company with representative offices in Atlanta (US), Sheffield (UK) and Würzburg (Germany); and development centers in Ukraine. Since 2009 it has grown from a small group of passionate engineers and business managers to a full-scale mobi...
With SaaS use rampant across organizations, how can IT departments track company data and maintain security? More and more departments are commissioning their own solutions and bypassing IT. A cloud environment is amorphous and powerful, allowing you to set up solutions for all of your user needs: document sharing and collaboration, mobile access, e-mail, even industry-specific applications. In his session at 16th Cloud Expo, Shawn Mills, President and a founder of Green House Data, discussed h...
For IoT to grow as quickly as analyst firms’ project, a lot is going to fall on developers to quickly bring applications to market. But the lack of a standard development platform threatens to slow growth and make application development more time consuming and costly, much like we’ve seen in the mobile space. In his session at @ThingsExpo, Mike Weiner, Product Manager of the Omega DevCloud with KORE Telematics Inc., discussed the evolving requirements for developers as IoT matures and conducte...
There are many considerations when moving applications from on-premise to cloud. It is critical to understand the benefits and also challenges of this migration. A successful migration will result in lower Total Cost of Ownership, yet offer the same or higher level of robustness. In his session at 15th Cloud Expo, Michael Meiner, an Engineering Director at Oracle, Corporation, analyzed a range of cloud offerings (IaaS, PaaS, SaaS) and discussed the benefits/challenges of migrating to each offe...
One of the hottest areas in cloud right now is DRaaS and related offerings. In his session at 16th Cloud Expo, Dale Levesque, Disaster Recovery Product Manager with Windstream's Cloud and Data Center Marketing team, will discuss the benefits of the cloud model, which far outweigh the traditional approach, and how enterprises need to ensure that their needs are properly being met.
In their session at 17th Cloud Expo, Hal Schwartz, CEO of Secure Infrastructure & Services (SIAS), and Chuck Paolillo, CTO of Secure Infrastructure & Services (SIAS), provide a study of cloud adoption trends and the power and flexibility of IBM Power and Pureflex cloud solutions. In his role as CEO of Secure Infrastructure & Services (SIAS), Hal Schwartz provides leadership and direction for the company.
In a recent research, analyst firm IDC found that the average cost of a critical application failure is $500,000 to $1 million per hour and the average total cost of unplanned application downtime is $1.25 billion to $2.5 billion per year for Fortune 1000 companies. In addition to the findings on the cost of the downtime, the research also highlighted best practices for development, testing, application support, infrastructure, and operations teams.
"We've just seen a huge influx of new partners coming into our ecosystem, and partners building unique offerings on top of our API set," explained Seth Bostock, Chief Executive Officer at IndependenceIT, in this SYS-CON.tv interview at 16th Cloud Expo, held June 9-11, 2015, at the Javits Center in New York City.
SYS-CON Events announced today that HPM Networks will exhibit at the 17th International Cloud Expo®, which will take place on November 3–5, 2015, at the Santa Clara Convention Center in Santa Clara, CA. For 20 years, HPM Networks has been integrating technology solutions that solve complex business challenges. HPM Networks has designed solutions for both SMB and enterprise customers throughout the San Francisco Bay Area.
The Software Defined Data Center (SDDC), which enables organizations to seamlessly run in a hybrid cloud model (public + private cloud), is here to stay. IDC estimates that the software-defined networking market will be valued at $3.7 billion by 2016. Security is a key component and benefit of the SDDC, and offers an opportunity to build security 'from the ground up' and weave it into the environment from day one. In his session at 16th Cloud Expo, Reuven Harrison, CTO and Co-Founder of Tufin,...
Mobile, social, Big Data, and cloud have fundamentally changed the way we live. “Anytime, anywhere” access to data and information is no longer a luxury; it’s a requirement, in both our personal and professional lives. For IT organizations, this means pressure has never been greater to deliver meaningful services to the business and customers.
Container technology is sending shock waves through the world of cloud computing. Heralded as the 'next big thing,' containers provide software owners a consistent way to package their software and dependencies while infrastructure operators benefit from a standard way to deploy and run them. Containers present new challenges for tracking usage due to their dynamic nature. They can also be deployed to bare metal, virtual machines and various cloud platforms. How do software owners track the usag...
The Internet of Everything (IoE) brings together people, process, data and things to make networked connections more relevant and valuable than ever before – transforming information into knowledge and knowledge into wisdom. IoE creates new capabilities, richer experiences, and unprecedented opportunities to improve business and government operations, decision making and mission support capabilities.
Chuck Piluso presented a study of cloud adoption trends and the power and flexibility of IBM Power and Pureflex cloud solutions. Prior to Secure Infrastructure and Services, Mr. Piluso founded North American Telecommunication Corporation, a facilities-based Competitive Local Exchange Carrier licensed by the Public Service Commission in 10 states, serving as the company's chairman and president from 1997 to 2000. Between 1990 and 1997, Mr. Piluso served as chairman & founder of International Te...
Puppet Labs has announced the next major update to its flagship product: Puppet Enterprise 2015.2. This release includes new features providing DevOps teams with clarity, simplicity and additional management capabilities, including an all-new user interface, an interactive graph for visualizing infrastructure code, a new unified agent and broader infrastructure support.