A Little More Science
Sangamo’s Factor IX product (SB-FIX) is the only one-time injection in vivo genome editing product currently in development for Hemophilia B. We hope 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 Factor IX product is specific for Hemophilia B, which inserts a working 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 SB-FIX may have the potential of expressing Factor IX protein for the life of the patient.
Animal models have shown the expression of normal human Factor IX protein in the blood after dosing with investigational SB-FIX. This is the first time SB-FIX will be used in humans.
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/genome-editing
Sangamo in vivo Genome Editing Product for Hemophilia B: www.sangamo.com/product-pipeline/hemophilia