USABiotech & Genomic Medicine▲
PTC Therapeutics to Acquire ST-920 Gene Therapy for Fabry Disease
PTC Therapeutics has agreed to acquire ST-920, a BLA-stage one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics in a $111 million upfront deal plus milestone payments. ST-920 is designed to deliver a functional copy of the GLA gene using an adeno-associated viral vector, and in the Phase 1/2 STAAR study patients showed sustained enzyme activity for up to 4.5 years alongside improvements in renal function, cardiac outcomes, and quality of life. The BLA submission for accelerated approval is based on the intermediate clinical endpoint of annualized eGFR at Week 52, with nonclinical and clinical modules already submitted and the CMC package expected in Q4 2026, targeting a 2027 launch. The therapy has FDA RMAT, Orphan Drug and Fast Track designations, and CEO Matthew B. Klein said the acquisition advances the company's strategy of leveraging its rare disease commercial infrastructure to accelerate near-term revenue growth. The acquisition is expected to close in Q3 2026, strengthening PTC's pipeline in rare genetic disorders.