PTC Therapeutics completes acquisition of Sangamo’s ST-920 Fabry gene therapy asset

PTC Therapeutics completed its acquisition of Sangamo’s ST-920, a BLA-stage one-time AAV gene therapy for Fabry disease. A rolling BLA filing to the FDA for accelerated approval is expected to be completed in Q4 2026. ST-920, also known as isaralgagene civaparvovec, targets long-term alpha-Gal A enzyme production to reduce Gb3 levels and cut reliance on ERT. The program holds FDA Orphan Drug, Fast Track, and RMAT designations, with similar designations in Europe and the UK.