Sangamo Therapeutics Presents Detailed Data from Registrational STAAR Study in Fabry Disease at WORLDSymposium™ 2026

Core Insights - The article highlights the potential of isaralgagene civaparvovec (ST-920) as a one-time, well-tolerated, and durable gene therapy for Fabry disease, which could significantly change the treatment landscape for this condition [1][3][5] Study Results - The STAAR study showed a positive mean annualized estimated glomerular filtration rate (eGFR) slope of 1.965 mL/min/1.73m²/year at 52 weeks across all 32 patients, indicating improved renal function [4] - Among 19 patients with 104 weeks of follow-up, the mean annualized eGFR slope was 1.747 mL/min/1.73m²/year, further supporting the therapy's efficacy [4] - Cardiac function remained stable over one year, with consistent cardiac structural stability observed across various clinical and demographic subgroups [4] Safety and Regulatory Pathway - Isaralgagene civaparvovec demonstrated a favorable safety profile without the need for preconditioning, making it a viable option for patients [4][5] - The FDA has agreed that the data from the STAAR study can serve as the primary basis for approval under the Accelerated Approval pathway, with a rolling submission of the Biologics License Application (BLA) initiated [1][6] Clinical Implications - The therapy shows potential to improve kidney function and stabilize cardiac health, addressing the multi-organ challenges posed by Fabry disease [3][5] - The ability to withdraw from current enzyme replacement therapy (ERT) is a significant advantage, offering a durable treatment option for patients [3][5] Future Presentations - Detailed data from the STAAR study will be presented at the 22nd Annual WORLDSymposium in San Diego, CA, from February 2-6, 2026, with multiple platform and poster presentations scheduled [2][12]