The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here.
Hemophilia B is a rare, genetic bleeding disorder caused by a lack of the factor IX (FIX) protein, which is necessary for normal blood clotting. Current hemophilia B treatments typically require frequent intravenous infusions of clotting FIX to minimize the number of bleeding episodes, but the burden on the patients is high and a risk of bleeding is always possible. SB-FIX uses Sangamo’s 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 FIX. 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 AAV cDNA gene therapy and lenti- or retroviral-based gene therapies that insert genes randomly into the genome, SB-FIX is designed with the goal to permanently and precisely integrate the F9 gene into the DNA.
“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. “This clinical trial will generate data highly anticipated by scientists, physicians and patients, and we are honored to be the first medical center to treat a patient.” Georgetown is conducting the study with its clinical partner, MedStar Georgetown University Hospital, where Kessler directs the Center for Comprehensive Hemophilia and Thrombosis Care.
Sangamo’s Phase 1/2 study is an open-label clinical trial designed to assess the safety, tolerability and preliminary efficacy of SB-FIX in adults with severe hemophilia B. The study is currently screening subjects in the United States at hospitals in Washington D.C., Duarte, Detroit, Indianapolis, Dallas, and in the United Kingdom in Glasgow, Birmingham and London.
“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. We’ve also initiated clinical sites in the United Kingdom and are currently screening patients for enrollment into this study.”
Read the press release in its entirety here.