RICHMOND, Calif., Sept. 14 /PRNewswire-FirstCall/ -- Sangamo BioSciences (Nasdaq: SGMO) will provide an update today to investors on the status of the latest developments in several of its ZFP Therapeutics programs. Scientists from the Company will provide details on programs focused on diabetic neuropathy (DN) and a new initiative for HIV therapy. Sangamo's proprietary technology is based on the engineering of a naturally occurring class of DNA binding proteins, zinc finger DNA-binding proteins (ZFPs). ZFPs can be engineered to create ZFP transcription factors (ZFP TFs) for therapeutic gene regulation and ZFP Nucleases (ZFNs) for targeted gene modification.
ZFP Therapeutic for Diabetic Neuropathy
Vascular endothelial growth factor (VEGF) has been shown to protect nerve cells and promote their growth. Sangamo has shown that a ZFP Therapeutic, SB 509, activates VEGF expression in cells and is applying this approach to the treatment of diabetic neuropathy. Cellular studies have demonstrated that SB 509 protects neuronal cells and in a rat model of diabetic neuropathy, a single treatment of this ZFP Therapeutic significantly improved both sensory and motor nerve conduction.
"We are on track to file an IND for SB 509 for the treatment of diabetic neuropathy before the end of this year," stated Edward Lanphier, Sangamo's president and CEO. "The Phase I/II trial is expected to enroll 25 patients and will evaluate the safety of SB 509 as well as the clinical effect on symptoms of peripheral neuropathy."
ZFP Therapeutic for HIV
In rare cases there are individuals who have been exposed multiple times to HIV-1, but who remain unaffected. These individuals appear to have inherited a shortened version of the gene for the CCR5 protein, which normally functions as the entry point for HIV into blood cells. The mutation in this gene results in a non-functional form of the CCR5 protein and makes individuals resistant to HIV infection.
Sangamo is using its ZFP technology to recreate this mutation in cells of the immune system in an attempt to protect cells against HIV infection. Key target cells for HIV, such as macrophages, dendritic cells and T-cells, can be targeted by Sangamo's approach which is designed to provide a reservoir of immune cells that can fight the opportunistic infections that are characteristic and often fatal in AIDS.
"This program demonstrates the versatility and breadth of our ZFP technology," said Dr. Dale Ando, Sangamo's vice president of therapeutic development and chief medical officer. "We believe that our approach is a fundamentally new strategy to address the pathology of HIV and AIDS and I look forward to leading our efforts to develop this application of our ZFN technology as a treatment for this terrible disease."
To access the webcast of the briefing, visit the Investor Relations section of the Sangamo website (www.sangamo.com) at http://phx.corporate-ir.net/phoenix.zhtml?c=120938&p=irol-IRHome . A replay of the webcast will be available online until September 28, 2004.
About Diabetic Neuropathy
Diabetic neuropathy affects approximately 50% of patients with diabetes, or roughly 6 million people in the United States. Left unchecked, diabetic neuropathy can lead to impaired sensation in the feet that can result in soft tissue damage, infection and ultimately amputation of the lower limb. In the USA in 2001, there were approximately 82,000 lower limb amputations performed on diabetics. There are currently no pharmaceutical therapies approved by the FDA for the treatment of diabetic neuropathy.
HIV, human immunodeficiency virus, is the virus that causes AIDS. The term AIDS applies to the most advanced stages of HIV infection. HIV progressively destroys the body's ability to fight infections and certain cancers. As of the end of 2003, an estimated 37.8 million people worldwide were living with HIV/AIDS. The Centers for Disease Control and Prevention (CDC) estimate that approximately 950,000 U.S. residents are infected with HIV and that there are approximately 40,000 new HIV cases each year in the U.S.
About Zinc Finger DNA Binding Proteins (ZFPs)
Zinc Finger Proteins (ZFPs) are a naturally occurring class of DNA binding proteins. The DNA recognition and binding function of ZFPs can be engineered and thus targeted to selected DNA sequences. 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 create ZFNs. ZFNs can be used to facilitate either the correction of mutant DNA sequences in genes as an approach to treating monogenic diseases or the disruption of genes that encode receptors for pathogens as an approach for infectious diseases.
Sangamo BioSciences, Inc is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification. The company's most advanced therapeutic development program is currently in a Phase I clinical trial and involves the use of a ZFP transcription factor for the treatment of peripheral artery disease. Other therapeutic development programs are focused on diabetic neuropathy, heart disease, cancer, neuropathic pain, and monogenic and infectious 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 TFs) 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 for a variety of monogenic diseases such as X-linked severe combined immunodeficiency (X-linked SCID) and sickle cell anemia (SCA) and 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, ZFNs, therapeutic applications of Sangamo's ZFP technology platform and the filing of an IND application and initiation of clinical trials. 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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