(MENAFN- GlobeNewsWire - Nasdaq) Single dose of avigbagene parvec (FLT201) demonstrates sustained clinical benefit and favorable safety up to two years after withdrawal of prior chronic therapy

Data underscore potential to set new standard-of-care

LONDON, Oct. 07, 2025 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced updated clinical data for avigbagene parvec (FLT201), its Gaucher disease gene therapy candidate, demonstrating clinical benefit and favorable safety maintained for more than two years to date following administration of a single low dose. These data are being presented at the European Society of Gene and Cell Therapy (ESGCT) 32^nd Annual Congress in Seville, Spain.

“People with Gaucher disease endure a heavy treatment burden, including lifelong biweekly infusions,” said Pilar Giraldo, MD, PhD, an investigator in the Phase 1/2 GALILEO-1 study of avigbagene parvec and a hematologist at Hospital Universitario Quironsalud, Zaragoza.“The data being presented at ESGCT show that a single infusion of avigbagene parvec enabled patients to remain off chronic therapy and maintain stable or improved clinical outcomes. These results highlight the potential of gene therapy to transform the care of Gaucher disease.”

The poster presentation highlights data from four patients with type 1 Gaucher disease in the Phase 1/2 GALILEO-1 trial who were taken off prior enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) after being treated with a single infusion of avigbagene parvec at the low dose of 4.5e11 vg/kg. All were taken off ERT or SRT within 11 weeks of dosing and remain off those therapies for between 19 and 23 months as of the data cutoff.

As of Aug. 25, 2025, the data showed:

• Rapid, robust and sustained reductions in glucosylsphingosine (lyso-Gb1), a measure of whole-body substrate build up and a validated biomarker of treatment response in Gaucher disease.
• Maintenance of normal hemoglobin levels.
• Improved or stable platelet counts.
• Stable liver and spleen volumes.
• Favorable safety and tolerability, with no dose-limiting toxicities.
  

Spur is preparing to initiate its planned Phase 3 trial of avigbagene parvec in the first half of 2026. The company has alignment with the US Food and Drug Administration on the design of a single-arm trial that could support both potential accelerated and full approval.

“Avigbagene parvec was specifically designed to address the shortcomings of the existing standard of care, with the aim of reducing both the disease and treatment burden for people with Gaucher,” said Pamela Foulds, MD, Chief Medical Officer of Spur Therapeutics.“These latest results build on the durability data we have previously reported, now showing favorable safety and efficacy for more than two years following a single low dose. As we prepare to move into the Phase 3 trial, we see tremendous potential for avigbagene parvec to set a new standard of care with a one-time gene therapy.”

About Spur Therapeutics
Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions. By optimizing every component of its product candidates, Spur aims to unlock the true potential of gene therapy to realize outsized clinical results. Spur is advancing a breakthrough gene therapy candidate for Gaucher disease and a preclinical gene therapy candidate for Parkinson's disease. Expanding our impact, and advancing the practice of genetic medicine.

Toward life-changing therapies, and brighter futures. Toward MoreTM

For more information, visit or connect with Spur on LinkedIn .

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