Sangamo Therapeutics Advances Rolling BLA for ST-920, Files Diagnostic PMA
Sangamo Therapeutics submitted preclinical and clinical modules in its rolling BLA to the FDA seeking accelerated approval for its one-time ST-920 gene therapy in adult Fabry disease. FDA agreed 52-week eGFR slope in STAAR study will support accelerated approval, and Sangamo filed its companion diagnostic with CDRH for PMA.
1. Rolling BLA Submission Progress
Sangamo Therapeutics began its rolling BLA submission in December 2025 and has now submitted both preclinical and clinical modules to the FDA for ST-920, enabling ongoing FDA review of completed sections rather than waiting for a full BLA file.
2. STAAR Study Endpoint Agreement
The Phase 1/2 STAAR study demonstrated a positive 52-week mean eGFR slope across all dosed adults with Fabry disease, which the FDA has agreed will serve as the primary endpoint to support accelerated approval.
3. Companion Diagnostic PMA Filing
Sangamo filed its antibody assay companion diagnostic with the FDA’s Center for Devices and Radiological Health seeking Premarket Approval to screen patients for eligibility for ST-920 infusion.