Sangamo Therapeutics’ updated phase 1/2 data continues to support the potential of its gene therapy candidate, isaralgagene civaparvovec (ST-920), as a one-time durable treatment option for Fabry disease, according to the company.
In the phase 1/2 STAAR study, 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. Sustained benefit was also demonstrated, with elevated expression of alpha-galactosidase A (α-Gal A) activity maintained for nearly four years for the longest treated patient as of the data cutoff date. Additionally, ST-920 was generally well tolerated with majority of AEs being grade 1-2 in nature.
The data was presented at the 21st Annual WORLDSymposium in California on February 6, 2025.
Sangamo expects to use the trial data to support an accelerated approval pathway in first half of 2025, with a potential BLA submission to the FDA anticipated in second half of 2025.
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. Isaralgagene civaparvovec is an investigational gene therapy using 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.
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