STEVENAGE, UK – FLT201, an experimental gene therapy developed by Spur Therapeutics, has shown sustained clinical benefits for up to 21 months in people with Gaucher disease type 1, according to new data from a clinical trial.
The findings, presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in New Orleans, come from the ongoing Phase 1/2 GALILEO-1 trial (NCT05324943).
“These findings highlight the power of our approach to set new standards of care for people with serious diseases,” said Dr. Pamela Foulds, chief medical officer at Spur Therapeutics.
What Is Gaucher Disease Type 1?
Gaucher disease type 1 is caused by mutations in the GBA1 gene, which lead to a deficiency in an enzyme responsible for breaking down fatty molecules called glucocerebrosides (Gb1). Without this enzyme, toxic buildup occurs in cells, causing symptoms such as:
- Enlarged liver and spleen
- Bone complications
- Blood abnormalities (e.g., low hemoglobin and platelet levels)
Unlike other forms, type 1 does not affect the nervous system.
How FLT201 Works
FLT201 is a one-time gene therapy that delivers a modified GBA1 gene via bloodstream infusion. The goal is to produce a more functional and stable enzyme capable of breaking down Gb1 and reducing toxic buildup.
Promising Early Trial Results
The GALILEO-1 trial enrolled six adults with Gaucher type 1, all of whom had been on standard therapies (enzyme replacement or substrate reduction) for at least two years. Each received a low dose of FLT201 (4.5×10¹¹ vector genomes/kg).
Among them, four participants discontinued standard treatments within three months after receiving FLT201. As of March, they remained off prior therapies for up to 21 months.
Key outcomes:
- Lyso-Gb1 levels, a biomarker of disease burden, remained low or continued to decline for up to 15 months post-treatment.
- One participant maintained low lyso-Gb1 levels 14 months after stopping therapy, despite already having well-managed levels.
- Hemoglobin levels remained within normal ranges.
- Platelet counts remained stable or improved for up to 18 months.
“These results show strong safety and efficacy signals nearly two years after a single dose,” said Dr. Foulds.
Additional Findings and Next Steps
Spur also presented preclinical data showing that FLT201 led to stable enzyme expression for more than 3.5 years in nonhuman primates.
Although some patients developed temporary antibodies to the enzyme post-treatment, Spur reported that these did not affect the therapy’s effectiveness.
Buoyed by these results, Spur plans to initiate a Phase 3 trial to further evaluate FLT201’s potential to reduce both disease and treatment burden for Gaucher patients.
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