Sangamo Therapeutics, Inc. recently announced it has treated the first patient in a Phase 1/2 clinical trial evaluating SB-FIX, the company’s investigational gene therapy for patients with hemophilia B. This is an open-label trial designed to assess the safety, tolerability and preliminary efficacy of SB-FIX in adults with severe hemophilia B and was granted Orphan Drug and Fast Track designations by the US Food and Drug Administration (FDA).
"I am grateful to the first patient entering this study as we explore the symbolic step toward potentially changing what treatment could look like for patients with hemophilia B," said study investigator Craig Kessler, MD, a professor of oncology at Georgetown University.
SB-FIX uses proprietary zinc finger nuclease (ZFN) genome editing to insert into the DNA of liver cells a corrective copy of the F9 gene, which controls the production of Factor IX. The goal of this therapy is to enable a patient's liver to produce a lifelong and stable supply of the clotting protein. Unlike conventional adeno-associated Virus (AAV) cDNA gene therapy and lenti- or retroviral-based gene therapies that insert genes randomly into the genome, SB-FIX is designed to permanently and precisely integrate the F9 gene into the DNA, according to Sangamo’s press release.
"We are excited to begin to understand the potential of our in vivo gene editing technology for hemophilia B, which represents a completely new treatment approach for this disease," said Sangamo chief medical officer, Edward Conner, MD. "We've made progress with patient recruitment in this clinical program and are hopeful for continued momentum in patient enrollment.
Source: Sangamo Therapeutics, Inc. press release dated December 17, 2018
