Sangamo, Pfizer Report Promising Interim Results With SB-525 Gene Therapy

(RTTNews) - Sangamo Therapeutics, Inc. (SGMO) and Pfizer, Inc. (PFE) announced interim data from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. The company said the data indicate that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII levels across the four dosage cohorts. Based on the results, the Safety Monitoring Committee recommended cohort expansion at the 3e13 vg/kg dose.

The U.S. FDA has granted Orphan Drug and Fast Track designations to SB-525, which also received Orphan Medicinal Product designation from the European Medicines Agency. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer.