You may qualify for the
FIXtendz clinical research
study if you:

 

Are a male at least 18 years old

  • Have severe hemophilia B and are currently free of inhibitor
  • Have no history of an allergic reaction to Factor IX (FIX) products

People who qualify will receive:

  • Investigational gene product (SB-FIX) for hemophilia B
  • Study-related medical care
  • Reimbursement for travel (Transportation assistance may be available)

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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

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