Sangamo Therapeutics, Inc. (SGMO), on Thursday, announced updated data from its Phase 1/2 STAAR study of isaralgagene civaparvovec or ST-920, a gene therapy designed to treat Fabry disease.
The results highlight the potential of ST-920 as a one-time, durable treatment that could significantly improve the lives of individuals suffering from this rare genetic disorder.
Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (a-Gal A) enzyme activity. This enzyme is required for metabolizing globotriaosylceramide (Gb3), and its deficiency leads to Gb3 buildup in cells, causing significant damage to vital organs such as the kidneys, heart, nerves, eyes, gut, and skin.
The latest data demonstrate sustained benefits from ST-920, including elevated levels of alpha-galactosidase A or a-Gal A activity, which were maintained for up to four years in the longest-treated patient. Additionally, the study revealed a positive mean estimated glomerular filtration rate or eGFR slope, suggesting improvements in kidney function—a key concern for Fabry disease patients.
Key Findings from the Updated Data:
-- The therapy maintained elevated a-Gal A activity for up to 47 months for the longest-treated patient, with up to 27 months of sustained benefit for those receiving the highest dose.
-- The study showed a positive mean annualized estimated glomerular filtration rate (eGFR) slope across 23 patients who had at least one year of follow-up, indicating significant improvements in renal function.
-- All 18 patients initially on enzyme replacement therapy or ERT successfully withdrew from ERT, with stable plasma lyso-Gb3 levels for up to 33 months in the longest-treated patient.
-- Notable improvements were observed in disease severity and quality of life, with a significant mean change of 10.6 points in the General Health score.
-- ST-920 was generally well-tolerated, with the majority of adverse events being mild or grade 1-2 and no serious adverse events, liver function test elevations, or deaths reported during the study.
Sangamo has received alignment from the FDA for an Accelerated Approval pathway for ST-920, with data from the Phase 1/2 STAAR study expected to serve as the basis for regulatory submission.
The company anticipates submitting a Biologics License Application to the FDA in the second half of 2025, following the availability of 52-week eGFR slope data for all enrolled patients in the first half of 2025.
Sangamo is also engaging in business development discussions to explore potential collaborations for the further advancement of ST-920.
Currently, SGMO is trading at $1.34 up by 6.34%.
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