FLT201 showed durable response and superior reduction of disease-causing substrate compared to ERT, underscoring its potential to improve upon standard of care
LONDON, May 27, 2025 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced the publication of preclinical proof-of-concept data for FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease in Molecular Therapy, the flagship journal of the American Society of Gene & Cell Therapy. These data supported the advancement of FLT201 into clinical development and highlight its potential to improve upon the current standard of care.
“FLT201 was designed to address the shortcomings of existing therapies for Gaucher disease,” said Pamela Foulds, M.D., Chief Medical Officer at Spur. “Many people with Gaucher disease continue to experience symptoms, including pain, fatigue and bone disease, despite lifelong, biweekly infusions of enzyme replacement therapy, or ERT. By delivering a more stable, rationally engineered version of the enzyme deficient in people with Gaucher disease, FLT201 has the potential provide better outcomes for patients with a single dose, dramatically reducing the burden of disease and the burden of treatment.”
The publication, titled “FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease Type 1,” highlights preclinical data showing that FLT201 outperformed both ERT and gene therapy delivering wildtype glucocerebrosidase (GCase).
Key findings from the publication include:
- Enhanced stability: GCase85, the rationally engineered version of GCase developed by Spur, demonstrated 6-fold longer half-life in serum and more than a 20-fold longer half-life at lysosomal pH than wildtype GCase in in vitro studies.
- Greater uptake in tissues: In a mouse model of Gaucher disease, this enhanced stability led to substantially higher GCase activity compared to ERT and wildtype GCase gene therapy. Notably, enzyme activity was observed in difficult-to-reach tissues, including bone and lung, which are poorly addressed by ERT.
- Superior substrate reduction: FLT201 also resulted in greater reductions of disease-causing substrates in affected tissues in this mouse model compared to ERT and wildtype GCase gene therapy.
- Durable expression. A single infusion of FLT201 resulted in robust increase in plasma GCase and sustained plasma GCase activity in non-human primates (NHPs), with expression maintained out to 3.5 years and no treatment-related adverse effects observed.
Data from the recently completed Phase 1/2 GALILEO-1 trial have shown a compelling safety and efficacy profile in adults with Gaucher disease following administration of a single low dose of FLT201. Spur is now preparing to advance FLT201 into a Phase 3 trial.
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, a potential first-in-class gene therapy candidate for adrenomyeloneuropathy and a preclinical gene therapy candidate for Parkinson’s disease, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of dementia and cardiovascular disease. Expanding our impact, and advancing the practice of genetic medicine.
Toward life-changing therapies, and brighter futures. Toward More™
For more information, visit www.spurtherapeutics.com or connect with Spur on LinkedIn.
Investor Contact
Naomi Aoki
naomi.aoki@spurtherapeutics.com
+ 1 617 283 4298
Media Contact
Carolyn Noyes
carolyn.noyes@spurtherapeutics.com
+ 1 617 780 2182
