RICHMOND, Calif., May 18, 2015 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced that new data from the company's proprietary ZFP Therapeutic® programs in disease models of lysosomal storage disorders (LSDs) were presented last week at the 18th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).
"These new data significantly 'de-risk' Sangamo's approach for Hunter and Hurler syndromes and potentially many other monogenic diseases, such as the hemophilias, that are currently treated using lifelong enzyme replacement therapy," stated Edward Lanphier, Sangamo's president and chief executive officer.
The data demonstrated durable expression of levels of functional enzyme sufficient to correct biomarkers of disease in mouse models of Hunter and Hurler syndromes, enabled by Sangamo's zinc finger nuclease (ZFN)-mediated genome editing technology.
The LSD programs are part of Sangamo's In Vivo Protein Replacement Platform (IVPRP) portfolio of ZFP Therapeutics. The IVPRP uses ZFN-mediated genome editing to precisely insert disease-corrective genes into the albumin locus in the genome of liver cells. The correct version of the enzyme, which is defective in a genetic disease, is then made by the powerful machinery that normally drives albumin expression, leading to the production and secretion of the missing enzyme by the liver.
A significant and sustained reduction in glycosaminoglycan (GAG) levels (a biomarker of lysosomal storage disorder progression) was observed in the urine and secondary tissue (liver, spleen, kidneys and lungs) in Hurler and Hunter mice 21 days post treatment with ZFNs. The accumulation of GAG oligosaccharides in the lysosomes of cells leads to dysfunction in several tissues throughout the body and is associated with symptoms of these LSDs. Production of the corrective enzyme was also shown to be durable through the twelve week study, consistent with earlier studies of the IVPRP for clotting factor, Factor IX, which demonstrated sustained production for over a year.
The data also demonstrated that in models of Hurler and Hunter syndrome, high levels of human IDUA (hIDUA) and human IDS (hIDS) enzyme activity respectively, could be detected in the liver (site of expression) and plasma of treated animals. In addition, functional enzymes could be detected in secondary tissues such as the spleen, kidneys, and lungs, demonstrating that they had been taken up into these tissues from the circulation.
"Our study demonstrates that the levels of enzyme present in various tissues were sufficient for the phenotypic correction of Hurler and Hunter syndromes mice, as measured by the reduction in GAG biomarkers, which suggests that the IVPRP can be successfully applied to these and potentially other LSDs," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "We are committed to advancing these programs through IND-enabling studies and into clinical trials as rapidly as possible to provide patients with potentially life-long protein replacement from a one-time therapy."
Additionally, at the meeting's Presidential Symposium on Friday, May 15, 2015, in one of four studies selected from all abstracts submitted to the conference, data were presented that demonstrated the success of a proprietary method for precise and highly efficient targeted gene addition in CD34+ hematopoietic stem and progenitor cells (HSPCs).
Finally, Dr. Philip Gregory, D.Phil., Sangamo's senior vice president of research and chief scientific officer, was one of three speakers invited to participate in the ASGCT Education Session, "Emerging Field Review: Gene Editing" on the final day of the meeting. His presentation covered recent technical developments made by Sangamo's scientists and collaborators in the field of genome editing using the research supporting Sangamo's HIV stem cell program and Sangamo and Shire's Huntington's disease program as examples.
Sangamo BioSciences, Inc. is focused on Engineering Genetic CuresTM for monogenic and infectious diseases by deploying its novel DNA-binding protein technology platform in therapeutic gene regulation and genome editing. The Company has a Phase 2 clinical program to evaluate the safety and efficacy of novel ZFP Therapeutics® for the treatment of HIV/AIDS (SB-728). Sangamo's other therapeutic programs are focused on monogenic and rare diseases. The Company has formed a strategic collaboration with Shire International GmbH to develop therapeutics for hemophilia, Huntington's disease and other monogenic diseases, and with Biogen Inc. for hemoglobinopathies, such as sickle cell disease and beta-thalassemia. It has also 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 contains forward-looking statements regarding Sangamo's current expectations. These forward looking statements include, without limitation, references to the research and development of novel ZFP TFs and ZFNs as ZFP Therapeutics and therapeutic application and the scope of such applications on Sangamo's ZFP technology platform to specific human diseases and unmet medical needs, including the development of ZFP Therapeutics for Hunter and Hurler syndromes and other monogenic diseases and stem cell applications.
These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the early stage of ZFP Therapeutic development, the lengthy and uncertain regulatory approval process, uncertainties related to the timing of initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of ZFP Therapeutics, and the ability to establish strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable gene-based therapeutics. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q as filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.
SOURCE Sangamo BioSciences, Inc.