Sangamo Therapeutics Reports Positive Fabry Disease Data, Neurology Pipeline Progress

Genomic medicine company advances gene therapy candidate, expands clinical-stage neurology portfolio

Mar. 31, 2026 at 3:38am

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A photorealistic studio still-life image featuring a polished, geometric glass vial filled with a vibrant blue liquid, symbolizing the promise of Sangamo's gene therapy technology. The vial is elegantly arranged on a clean, monochromatic background, using dramatic lighting and shadows to convey the scientific innovation behind the company's work.Sangamo's gene therapy technology aims to deliver transformative treatments for patients with rare genetic disorders and neurological diseases.Richmond Today

Sangamo Therapeutics, a genomic medicine company, reported recent business highlights and financial results for the fourth quarter and full year 2025. Key updates include positive topline data from the registrational STAAR study of its Fabry disease gene therapy candidate, progress in the rolling BLA submission, and the expansion of its clinical-stage neurology pipeline.

Why it matters

Sangamo's Fabry disease gene therapy has the potential to provide a one-time, durable treatment option that could fundamentally shift the treatment paradigm for this rare genetic disorder. The company's transition to a clinical-stage neurology company also marks an important milestone as it seeks to address devastating neurological diseases with limited treatment options.

The details

Sangamo reported positive topline results from the registrational STAAR study of its Fabry disease gene therapy candidate, isaralgagene civaparvovec. The data showed a positive mean annualized eGFR slope at 52 weeks across all dosed patients, indicating improved renal function. The company also reported stable cardiac function and durable enzyme expression over time. Sangamo is advancing the rolling BLA submission for isaralgagene civaparvovec, expected as early as summer 2026. In its neurology pipeline, Sangamo has activated six clinical sites for the Phase 1/2 STAND study of ST-503 in chronic neuropathic pain and is progressing CTA-enabling activities for ST-506 in prion disease.

  • In December 2025, Sangamo initiated a rolling submission of a BLA to the FDA for isaralgagene civaparvovec in Fabry disease.
  • In February 2026, Sangamo presented detailed data from the STAAR study at the WORLD Symposium.
  • In March 2026, a manuscript on the preclinical safety and pharmacology of ST-503 was published in Science Translational Medicine.

The players

Sangamo Therapeutics

A genomic medicine company dedicated to developing treatments for serious neurological diseases.

Sandy Macrae

Chief Executive Officer of Sangamo Therapeutics.

ST-920

Sangamo's gene therapy product candidate for the treatment of Fabry disease.

ST-503

Sangamo's investigational epigenetic regulator for the treatment of chronic neuropathic pain.

ST-506

Sangamo's investigational epigenetic regulator for the treatment of prion disease.

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What they’re saying

“Sangamo continued to make significant pipeline progress since the start of 2025. Following positive topline results from our registrational STAAR study in Fabry disease, we are well advanced in the rolling submission of the BLA to the FDA under the Accelerated Approval pathway.”

— Sandy Macrae, Chief Executive Officer

What’s next

Sangamo expects to complete the rolling BLA submission for isaralgagene civaparvovec in Fabry disease as early as the summer of 2026, subject to securing adequate additional funding.

The takeaway

Sangamo's progress in its Fabry disease program and the expansion of its clinical-stage neurology pipeline demonstrate the company's commitment to developing transformative genomic medicines for patients with serious neurological disorders who lack adequate treatment options.