PTC completes acquisition of ST-920 Fabry gene therapy from Sangamo; BLA submission expected Q4 2026

PTC THERAPEUTICS, INC.

Completed acquisition of ST-920, a BLA-stage one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics.
Rolling BLA submission to FDA for accelerated approval expected to be completed in Q4 2026.
BLA based on 52-week Phase 1/2 STAAR study showing favorable renal function effect and safety/tolerability.
Company also plans to pursue regulatory approval of ST-920 outside the United States.
CEO Matthew Klein cites potential to address limitations of chronic enzyme replacement therapy for Fabry community.

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