A Little More Science
Sangamo’s Factor IX product (SB-FIX) is the only one-time treatment in vivo genome editing therapy currently in development for Hemophilia B. We expect that Sangamo’s proprietary ZFN-mediated genome editing approach will provide enhanced durability, with no washout or decrease in constitutive Factor IX expression over time as compared to first-generation gene therapy approaches.
Sangamo’s therapeutic Factor IX product is specific for Hemophilia B, which inserts a therapeutic version of the Factor 9 gene into liver cells to drive constitutive Factor IX protein expression. As the correct gene is integrated into the patient’s liver cells, our therapy may have the potential of expressing Factor IX protein for the life of the patient.
Studies have shown that the use of this treatment in mouse models induces therapeutic levels of expression of a normal human Factor IX protein, and that this Factor IX protein corrects bleeding time in treated animals to normal levels.
In addition, treatment of non-human primates with Sangamo’s approach produced therapeutic levels of Factor IX in the blood. This positive preclinical proof of concept data provided critical support to move into the clinic with SB-FIX in Hemophilia B.
For more information, please visit the following links.
Journal article: www.bloodjournal.org/content/bloodjournal/126/15/1777.full.pdf
Sangamo Genome Editing Technology: www.sangamo.com/technology/zf-nucleases.html
Sangamo in vivo Genome Editing Product for Hemophilia B: www.sangamo.com/pipeline/hemophilia.html