Sangamo Therapeutics, Inc. (SGMO), a genomic medicine firm, on Tuesday announced positive data from the registrational Phase 1/2 STAAR study evaluating its lead candidate, isaralgagene civaparvovec, or ST-920, for the treatment of Fabry disease.
The promising results of ST-920 from the registrational Phase 1/2 STAAR study include a positive mean annualised estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients in the study, stable cardiac function over one year, and durability of effect, with elevated expression of alpha-galactosidase A (a-Gal A) activity maintained for up to 4.5 years.eGFR estimates kidney function in filtering waste.
These data will be presented in four platform presentations and in poster sessions at the 22nd Annual WORLD Symposium, taking place in San Diego, CA, February 2-6, 2026.
Following the news, SGMO is up 7.74% in the overnight market at $0.42.
Fabry disease is a rare genetic disorder classified as an X-linked lysosomal storage disease. People with this condition lack or have insufficient amounts of an enzyme called alpha-galactosidase A (a-Gal A). This deficiency leads to the buildup of certain fatty substances in various organs, causing a wide range of symptoms and complications.
Isaralgagene Civaparvovec, also known as ST-920, is Sangamo's innovative gene therapy being studied in clinical trials for the treatment of Fabry Disease.
The STAAR study enrolled patients who were on Enzyme Replacement Therapy (ERT), ERT pseudo-naïve defined as having been off ERT for six or more months, or ERT-naïve. The Phase 1/2 STAAR study is complete, and 32 patients have successfully rolled into the long-term follow-up study.
As of the April 10, 2025, data cut-off date, a positive mean annualized eGFR slope of 1.965 mL/min/1.73m2/year at 52-weeks was observed across all 32 dosed patients, indicating an improvement in renal function.
Furthermore, a mean annualised eGFR slope of 1.747 mL/min/1.73m2/year (95% CI: -0.106, 3.601) was observed for the 19 patients who had achieved 104 weeks of follow-up.
Stable cardiac function was observed over one year, including consistent cardiac structural stability across clinical and demographic subgroups.
Also, the company noted that the durability of effect was demonstrated with elevated expression of alpha-galactosidase A (a-Gal A) activity maintained for up to 4.5 years for the longest treated patient, with other clinical benefits.
Isaralgagene civaparvovec demonstrated a favorable safety and tolerability profile in the study, without the requirement for preconditioning.
The FDA has granted Orphan Drug, Fast Track, and RMAT designations to isaralgagene civaparvovecfor Fabry disease. The FDA provided a regulatory pathway to accelerated approval for isaralgagene civaparvovec, agreeing that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the Accelerated Approval Program, using mean annualized eGFR slope at 52 weeks as an intermediate clinical endpoint.
In December 2025, Sangamo initiated a rolling submission of a BLA to the FDA seeking approval of isaralgagene civaparvovec under an Accelerated Approval pathwayfor Fabry Disease.
According to Grandview Research, the global Fabry disease treatment market was valued at $2.54 billion in 2023 and is projected to reach $4.93 billion by 2030, growing at a 9.9% CAGR from 2024 to 2030.
"We are pleased to have initiated a rolling submission of a BLA to the FDA seeking approval of ST-920," said Nathalie Dubois-Stringfellow, Ph.D, Chief Development Officer at Sangamo.
SGMO closed Tuesday's trade at $0.39, down 31.33%.
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