Sangamo Therapeutics and Pfizer announced the first patient received treatment in the Phase 1/2 clinical trial (Alta trial) evaluating SB-525, an investigational gene therapy for patients with Hemophilia A, a rare genetic blood disorder.

"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.

The Alta trial is an open-label, adaptive, dose-ranging clinical study designed to assess the safety and tolerability of SB-525 investigational gene therapy in up to 20 adult subjects with severe Hemophilia A.  SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer for the development and commercialization of gene therapy programs for Hemophilia A.

"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."

SB-525 comprises a recombinant adeno-associated virus (rAAV) vector carrying a Factor VIII gene construct driven by a proprietary, synthetic, liver-specific promoter. The U.S. Food and Drug Administration has granted Orphan Drug and Fast Track designations to SB-525, which also received Orphan Medicinal Product designation from the European Medicines Agency. Hemophilia A is a rare blood disorder caused by a genetic mutation resulting in insufficient activity of Factor VIII, a blood clotting protein the body uses to stop bleeding. There are approximately 16,000 patients in the U.S. and more than 150,000 worldwide with Hemophilia A.