RICHMOND, Calif. — Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has accepted Sangamo’s request for a rolling submission and review of the Biologics License Application (BLA) for isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease.
This acceptance follows Sangamo’s meeting with the FDA in October 2025 to discuss the proposed efficacy and safety data package for isaralgagene civaparvovec where, in the meeting minutes, among other things, the FDA reiterated its October 2024 agreement to use eGFR slope as an endpoint to support an accelerated approval pathway.
“We are pleased to have received acceptance of our rolling submission and review request from the FDA, which follows our recent meeting to discuss the proposed efficacy and safety data package,” said Nathalie Dubois-Stringfellow, Ph. D, Chief Development Officer at Sangamo. “We are excited by the potential of ST-920 to provide a potentially transformative treatment for Fabry disease patients and look forward to initiating rolling submission of the BLA later this quarter.”
Sangamo presented detailed clinical data from the registrational Phase 1/2 STAAR study at the International Congress of Inborn Errors of Metabolism 2025 (ICIEM2025) in September, which demonstrated the potential for isaralgagene civaparvovec as a one-time, durable treatment of underlying pathology of Fabry disease to provide meaningful, multi-organ, clinical benefits above current standards of care. Furthermore, the STAAR study demonstrated a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which the FDA has agreed will serve as the primary basis of approval.
Isaralgagene civaparvovec has been granted Orphan Drug, Fast Track and RMAT designations from the FDA, Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from U.K. Medicines and Healthcare products Regulatory Agency.
Sangamo plans to initiate rolling submission of the BLA to the FDA under the accelerated approval pathway later in the fourth quarter of 2025.
About the STAAR Study
The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities.
About Sangamo Therapeutics
Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders. Moreover, Sangamo’s SIFTER capsid discovery platform is advancing delivery to the central nervous system in preclinical studies. Sangamo is also progressing next generation genome editing through its modular integrase (MINT) platform. Sangamo’s pipeline includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit www.sangamo.com and connect with us on LinkedIn and Twitter/X.
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