Sangamo reports positive results for Fabry disease gene therapy

RICHMOND, Calif. - Sangamo Therapeutics, Inc. (NASDAQ:SGMO), a clinical-stage biotechnology company with a market capitalization of $103 million and trailing twelve-month revenue of $64 million, announced positive topline results from its registrational Phase 1/2 STAAR study of isaralgagene civaparvovec, a gene therapy for adults with Fabry disease. According to InvestingPro data, the company currently trades below its Fair Value despite challenging financials.

The 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 dosed patients. This endpoint, which the FDA has agreed will serve as the primary basis for approval under the Accelerated Approval pathway, contrasts with the declining eGFR slopes typically seen with existing treatments. The company’s stock, which has declined over 80% in the past six months, could see renewed interest following these positive results.

Patients who reached 104 weeks of follow-up maintained a positive mean annualized eGFR slope of 1.747 mL/min/1.73m²/year. The company noted that published studies of current treatments show estimated mean annualized eGFR slopes ranging from -2.2 to -0.4 mL/min/1.73m²/year.

All 18 patients who began the study on enzyme replacement therapy have discontinued that treatment and remain off it. The therapy also demonstrated elevated expression of alpha-galactosidase A activity maintained for up to 4.5 years in the longest-treated patient.

The treatment showed a favorable safety profile without requiring preconditioning. Most adverse events were mild to moderate, with the most common being fever, COVID-19, headache and nasopharyngitis.

Based on these results, Sangamo plans to submit a Biologics License Application to the FDA in 2026.

Fabry disease is a lysosomal storage disorder caused by mutations in the GLA gene, leading to deficient enzyme activity that can damage vital organs including the kidneys, heart, nerves and skin.

The information in this article is based on a company press release statement.

In other recent news, Sangamo BioSciences has been actively engaging in strategic developments. The company reported its Q1 2025 earnings, highlighting a significant collaboration with Eli Lilly, which could bring up to $1.4 billion in milestone payments. Despite this, the lack of immediate financial results or guidance revisions led to a notable drop in stock value. The company has also secured $23 million in financing, aimed at extending its financial runway and supporting ongoing negotiations for its Fabry disease program. Analysts from Jefferies have revised their price target for Sangamo, lowering it to $1.50 while maintaining a Buy rating, indicating cautious optimism about the company’s future. Meanwhile, H.C. Wainwright continues to maintain a Buy rating with a $10 target, citing confidence in Sangamo’s short-term strategy and upcoming milestones. Sangamo is preparing for significant upcoming events, including a potential Biologics License Application for its Fabry disease treatment in 2026. Additionally, the company’s pipeline advancements in treatments like ST-503 and ST-506 are progressing, with preliminary results expected in late 2026.

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