Sangamo Therapeutics, Inc. and Pfizer Inc. recently announced that the first patient received treatment in a phase 1/2 clinical trial designed to evaluate SB-525, the company’s gene therapy program for people with hemophilia A, or factor VIII (FVIII) deficiency. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer for the development and commercialization of hemophilia A gene therapy programs.
The trial is an open-label, dose-ranging clinical study designed to assess the safety and tolerability of SB-525 in up to 20 adults with severe hemophilia A. The investigational therapy is developed using adeno-associated viruses (AAVs) as delivery vehicles, or vectors, to carry the genetic codes that initiate the production of the deficient factor protein—in this case, FVIII in those with hemophilia A. The AAVs deliver this genetic material into liver cells, without causing disease or triggering significant immune responses.
“We are excited to announce the first patient infusion in this initial clinical trial of SB-525. Gene therapy holds promise as a potential one-time treatment to control hemophilia A and prevent spontaneous bleeding,” said Dr. Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics.
“We are excited to see progress in our partnership program for SB-525 with Sangamo,” said Greg LaRosa, Chief Scientific Officer, Pfizer Rare Disease. “By combining Sangamo’s scientific and technical expertise across multiple genomic platforms, with Pfizer's global reach, resources and expertise, we are advancing the promise of gene therapy with the hope of making a meaningful impact for people living with hemophilia A.”
Source: Sangamo press release dated August 25, 2017
