RICHMOND, Calif., May 4 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. , the world leader in the research and development of zinc finger DNA-binding proteins (ZFPs), announced today that it has received a US$100,000 Grand Challenges Explorations grant from the Bill & Melinda Gates Foundation. The grant will support an innovative global health research project conducted by Sangamo scientists and titled “Zinc Finger Nucleases for In Vivo Treatment of HIV Infection.”
Sangamo’s project is one of 81 grants announced by the Gates Foundation in the second funding round of Grand Challenges Explorations, an initiative to help scientists around the world explore bold and largely unproven ways to improve health in developing countries. The grants were provided to scientists in 17 countries on six continents.
To receive funding, the company showed in a two-page application how its idea falls outside current scientific paradigms and might lead to significant advances in global health. The initiative is highly competitive, receiving more than 3,000 proposals in this round.
People born with a genetic mutation in their CCR5 gene, a critical receptor for HIV entry, are naturally resistant to HIV infection. Sangamo has designed and engineered zinc finger DNA-binding protein nucleases (ZFN(TM)) that specifically disrupt the CCR5 gene in cells. In preclinical work published with their collaborators at the University of Pennsylvania, Sangamo researchers demonstrated that in an animal model of HIV infection ZFN-modified human immune cells survived and multiplied preferentially, leading to an increase in CD4+ T-cell counts and a reduction in viral load suggesting resistance to HIV (Nat Biotechnol. 2008 Jul; 26(7):808-16). A Phase 1 clinical trial is ongoing at the University of Pennsylvania to evaluate the safety and tolerability of SB-728-T, a ZFP Therapeutic based on Sangamo’s CCR5-disrupting ZFNs. SB-728-T is being tested as an ex-vivo, or cell-therapy, approach for the treatment of HIV/AIDS.
Sangamo proposes to develop the next generation of this therapeutic approach, an in-vivo, or direct injection protocol. The resulting CCR5-negative cells will be protected from HIV infection and have the potential to provide long term control of the opportunistic infections characteristic of AIDS as well as HIV itself. Ultimately this approach could be combined with traditional vaccination strategies to further arm the immune system against HIV infection.
“We are very pleased that our research proposal was chosen for this prestigious award,” commented Edward Lanphier, Sangamo’s president and CEO. “We already have an ongoing Phase 1 clinical trial to evaluate our ZFN-based approach as an ex-vivo formulation. This grant will aid our efforts to develop the next generation of this ZFP Therapeutic, an in-vivo formulation for direct injection that could make our approach accessible to HIV-infected patients throughout the world.”
“The winners of these grants are doing truly exciting and innovative work,” said Dr. Tachi Yamada, president of the Gates Foundation’s Global Health Program. “I’m optimistic that some of these exploratory projects will lead to life-saving breakthroughs for people in the world’s poorest countries.”
About Grand Challenges Explorations
Grand Challenges Explorations is a five-year, $100 million initiative of the Gates Foundation to promote innovation in global health. The program uses an agile, streamlined grant process - applications are limited to two pages, and preliminary data are not required. Proposals are reviewed and selected by a committee of foundation staff and external experts, and grant decisions are made within approximately three months of the close of the funding round.
Applications for the next round of Grand Challenges Explorations are being accepted through May 28, 2009. Grant application instructions, including the list of topic areas in which proposals are currently being accepted, are available at the Grand Challenges Explorations website.
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 program is currently in Phase 2 clinical trials for evaluation of safety and clinical effect in patients with diabetic neuropathy and ALS. Sangamo also has a Phase 1 clinical trial to evaluate safety and clinical effect of a ZFP Therapeutic for the treatment of HIV/AIDS. Other therapeutic development programs are focused on cancer, neuropathic pain, nerve regeneration, Parkinson’s disease 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 (ZFN(TM)) for gene modification. Sangamo has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences, Sigma-Aldrich Corporation and several companies applying its ZFP technology to engineer cell lines for the production of protein pharmaceuticals. For more information about Sangamo, visit the company’s web site at www.sangamo.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, human, plant and research applications of Sangamo’s ZFP technology platform, strategic partnerships with collaborators and clinical trials of ZFP Therapeutics. 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 report on Form 10-Q. Sangamo BioSciences, Inc. assumes no obligation to update the forward-looking information contained in this press release.
CONTACT: Elizabeth Wolffe, Ph.D. of Sangamo BioSciences, Inc.,
+1-510-970-6000, x271, ewolffe@sangamo.com
Web site: http://www.sangamo.com/
