Dec. 10, 2012
/PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of new pre-clinical data demonstrating the successful application of its In Vivo Protein Replacement Platform. Based on Sangamo's zinc finger DNA-binding protein (ZFP) genome-editing technology, the platform enables the permanent production of therapeutic proteins from the liver with a single systemic treatment, potentially providing curative treatments for a range of monogenic diseases including hemophilia and lysosomal storage diseases (LSD) such as Gaucher, and Fabry disease. Such diseases are currently treated by regular infusions of protein or enzyme replacement therapy (ERT) throughout the patient's life. The data were presented at the 54
Annual Meeting of the American Society of Hematology (ASH) which is being held in
"These data provide proof-of-concept for this broadly applicable genome editing strategy," said
. D. Phil., Sangamo's vice president of research and CSO. "We show that a single systemic treatment enables stable liver-specific production of human Factor IX protein, the clotting factor absent in hemophilia B, reaching or exceeding 100% of normal circulating levels. In addition, our data in multiple lysosomal storage diseases serve to demonstrate the potential of this approach for a broad range of other monogenic diseases."
Sangamo's In Vivo Protein Replacement Platform makes use of a highly expressed and liver specific genomic "safe-harbor site" that can be edited with ZFP nucleases (ZFNs) to accept and express any therapeutic gene and thus permanently produce high levels of the missing protein with a single treatment. The gene encoding albumin, the most abundant protein in blood serum, was chosen as a safe harbor site because it is highly expressed exclusively in the liver, continuously producing large amounts of albumin protein (approximately 15g/day). With such a large capacity for protein production, targeting and co-opting a very small percentage of the body's albumin production capacity is sufficient to safely produce the needed replacement protein at therapeutically relevant levels. The study was performed in collaboration with the laboratory of
Katherine A. High
, M.D., director of the Center for Cellular and Molecular Therapeutics at The Children's Hospital of
. Dr. High, a Howard Hughes Medical Institute Investigator, is also a Professor of Pediatrics at the Perelman School of Medicine at the
University of Pennsylvania
"This platform demonstrates our ability to leverage our ZFN technology across multiple disease areas and enables us to rapidly expand our ZFP Therapeutic pipeline," stated
, Sangamo's president and chief executive officer. "As these presentations at ASH demonstrate, our ZFN gene-editing platform has broad utility with the potential to provide a disruptive and broadly leverageable therapeutic approach to a variety of diseases with unmet medical needs. Our In Vivo Protein Replacement Platform has the potential to impact any disease-relevant gene where enabling the liver to provide a stable source of corrective replacement protein will be therapeutic."
The data were presented in an oral presentation: