Sangamo Therapeutics has advanced the rolling submission of its BLA seeking accelerated approval of isaralgagene civaparvovec (ST-920), an 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.
Sangamo believes that the totality of data from the registrational STAAR study demonstrates the potential of ST-920 as a one-time, well-tolerated and durable gene therapy treatment option that can provide meaningful, multi-organ clinical benefits that could fundamentally shift the Fabry treatment paradigm.
In February, Sangamo shared updated data from the phase 1/2 STAAR study where positive mean estimated glomerular filtration rate (eGFR) slope was observed in the 23 patients who had reached at least one-year follow-up, indicating notable improvements in kidney function — an important predicter of mortality in Fabry disease. Now, according to Sangamo, the FDA has agreed that the study’s positive mean annualized eGFR slope at 52-weeks will serve as endpoint to support accelerated approval.
Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient 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. ST-920 uses a recombinant AAV2/6 vector containing human GLA cDNA designed to produce continuous, liver-specific α-Gal A expression. The treatment requires a one-time infusion without preconditioning.
Subscribe to our e-Newsletters
Stay up to date with news, articles and insights relevant to cell and gene therapy development and manufacturing. Plus, get special offers from Cell & Gene Therapy Review delivered right to your inbox!
Sign up now!