Sangamo BioSciences Provides Comprehensive Overview Of Company's ZFP Therapeutic® Development Programs At Its 2012 Analyst BriefingCompany Introduces Novel "In Vivo Protein Replacement Platform" Leverageable Across Many Monogenic Diseases Potentially Enabling Multiple New INDs by the End of 2015
NEW YORK, Dec. 6, 2012 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO), announced that the company is providing an update on its technology platform advancements and pipeline of ZFP Therapeutics® as well as near- and mid-term operating goals during an Analyst and Investor Briefing being held today in New York City. The presentation, by members of Sangamo management team, will be webcast live beginning at 5:00 pm ET and can be accessed via a link on the Company's website.
"We are very excited to share the progress that we have made in advancing our zinc finger DNA binding protein (ZFP) technology and ZFP Therapeutic programs, particularly in the area of monogenic diseases where we have developed a new, highly disruptive therapeutic platform for protein replacement therapies," said Edward Lanphier, president and CEO of Sangamo. "This approach can be leveraged across multiple diseases with the goal of engineering genetic cures."
"Sangamo's proprietary gene-editing and gene-regulation technology is unique in its ability to generate novel, highly differentiated therapies that act at the DNA-level," stated Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "This capability enables us to address the source of a wide variety of genetic diseases and potentially cure them. In addition to two ongoing Phase 2 clinical trials of SB-728-T, which may afford a functional cure for HIV/AIDS, we have a rich pipeline of preclinical programs. These include ZFP Therapeutics for hemophilia and Huntington's disease, which we are developing in collaboration with Shire, as well as Sangamo's programs in hemoglobinopathies (sickle cell disease and beta-thalassemia) and lysosomal storage diseases (LSDs). Based upon our ZFP Therapeutic approach and the disease targets that we have selected, we expect to file up to seven new INDs by 2015."
"Commercially, our business model has enabled us to monetize the value of our ZFP technology platform and maintain a strong balance sheet through partnerships with Dow AgroSciences in plant agriculture and Sigma-Aldrich Corporation in life science research reagents, while making significant progress in advancing our ZFP Therapeutics programs," continued Mr. Lanphier. "Beyond our very significant collaboration with Shire, we plan to establish additional high-value strategic partnerships around selected therapeutic programs at points of realizable value inflection while continuing to advance proprietary programs and increasingly forward integrate."
Program Highlights
During the briefing members of Sangamo's management team will discuss recent achievements and objectives for the 2013-2015 time period including:
Clinical
Sangamo plans to present preliminary data in the first half of 2013 from its clinical trials of SB-728-T (SB-728-1101 and SB-728-902 Cohort 5) for the treatment of HIV/AIDS and expects to have the full data set from these trials by the end of 2013. Both studies are designed to maximize the engraftment of T-cells in which both copies of the CCR5 gene have been disrupted making the cells resistant to infection by HIV. This creates a reservoir of cells that cannot be infected by the virus but are available to mount an immune response with the goal of providing a "functional cure" for HIV.Preclinical / Research
Sangamo will outline its strategy to enable the potential filing of seven Investigational New Drug (IND) Applications by the end of 2015.The Company will introduce a new, highly disruptive "In Vivo Protein Replacement Platform" driven by Sangamo's ZFP nuclease (ZFN) technology which is potentially curative and leverageable across many monogenic diseases, such as hemophilia and LSDs (e.g. Gaucher, Fabry, and Pompe disease) that are currently treated by regular infusions of enzyme replacement therapy (ERT). Sangamo's novel approach uses a naturally highly expressed gene to drive continuous production of active forms of these proteins from the liver such that they are present at stable levels in the bloodstream thus reducing or eliminating the need for ERT.The Company will update investors on recent preclinical progress in ZFP Therapeutics programs with the goal of engineering a genetic cure for hemophilia, Huntington's disease and hemoglobinopathies (sickle cell disease and beta-thalassemia) and outline the key next steps and timelines to IND filing.
Financial
Sangamo will provide a near- and mid-term financial overview including the anticipated amount of cash and cash equivalent of $40-$45 million by the end of 2015 assuming completion of milestones associated with existing partnerships but not including any additional funding from new partnerships, research grants or equity financing transactions.Sangamo's 2012 Analyst and Investor Briefing is being webcast live and will be available on Sangamo's website at 5:00pm ET via a link in the Investor section at investor.sangamo.com under "Events and Presentations". A replay of the webcast will be archived and available on the website approximately two hours after the presentation. |
