7:10AM Sangamo BioSci presents data from non-human primates providing proof of concept for disruptive and broadly leverageable platform for Protein Replacement Therapies; Therapeutic Levels of gene modification in Sangamo's In Vivo Protein Replacement Platform (SGMO) 12.40 : Co announced the presentation of new pre-clinical data demonstrating therapeutic levels of gene modification in non-human primates (NHPs) from 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 a specific genomic site in the liver with a single systemic treatment, potentially providing curative treatments for a range of monogenic diseases such as hemophilia and lysosomal storage disorders (LSD) including 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 55th Annual Meeting of the American Society of Hematology (ASH) which is being held in New Orleans.
"These data provide proof of concept for this broadly applicable genome editing strategy by demonstrating that our process is scalable to large animals and by validating the use of the albumin safe harbor as a site for expression of therapeutic proteins," said Philip Gregory. D. Phil., Sangamo's vice president of research and chief scientific officer. "We have further optimized Sangamo's ZFN system and demonstrated that a single systemic treatment enables stable liver-specific production of replacement protein. Early data in primates suggest that we can achieve circulating levels of protein above the threshold for therapeutic effect, which we believe are sufficient for the correction of a range of monogenic diseases. Our data demonstrate expression of replacement enzymes for multiple different proteins, including those deficient in lysosomal storage disorders, which serves to demonstrate the potential of this approach for a broad range of other monogenic diseases."
