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Sangamo Therapeutics Advances Rolling Submission of BLA to U.S. FDA for ST-920 in Fabry Disease
Data support potential of isaralgagene civaparvovec as a one-time, well tolerated and durable Fabry disease gene therapy to provide meaningful, multi-organ
About this update from Sangamo Therapeutics, Inc.
[{"type":"text","content":"Data support potential of isaralgagene civaparvovec as a one-time, well tolerated and durable Fabry disease gene therapy to provide meaningful, multi-organ clinical benefits that could fundamentally shift Fabry treatment paradigm STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which U.S. Food and Drug Administration (FDA) has agreed will serve as an endpoint to support accelerated approval pathway RICHMOND, Calif., March 09, 2026 (GLOBE NEWSWIRE) -- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced advancement of the rolling submission of a BLA to the FDA seeking accelerated approval of isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease. Following initiation of the rolling submission in December 2025, Sangamo has now submitted the preclinical and clinical modules to the FDA for review. Rolling submission allows for completed modules of the BLA to be submitted and reviewed by the FDA on an ongoing basis rather than waiting for the entire BLA to be submitted at once. In addition, the antibody assay companion diagnostic, which is designed to screen patients for eligibility with isaralgagene civaparvovec, has been submitted to, and accepted by, the FDA’s Center for Devices and Radiological Health (CDRH) seeking Premarket Approval (PMA). Sangamo believes that the totality of data from the registrational STAAR study demonstrates the potential of isaralgagene civaparvovec as a one-time, well-tolerated and durable gene therapy treatment option for Fabry disease to provide meaningful, multi-organ clinical benefits that could fundamentally shift the Fabry treatment paradigm. Furthermore, the STAAR study demonstrated a positive mean annualized eGFR slope at 52-weeks across all dosed patients in the study, which the FDA has agreed will serve as endpoint to support accelerated approval. These data were presented via four platform presentations and in poster presentations at the recent 22nd Annual WORLDSymposium™. These data are available on Sangamo’s website on the Presentations page. About the STAAR StudyThe Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluat...