Spur Therapeutics to Present Two-Year Clinical Data for FLT201 Gene Therapy in Gaucher Disease at WORLDSymposium
Key Insights
Spur Therapeutics (search) will present updated two-year clinical data from Phase 1/2 GALILEO-1 and GALILEO-2 trials of avigbagene parvec (search) (FLT201) for Type 1 Gaucher disease (search) at the 22nd Annual WORLDSymposium.
The presentations will include biomarker and clinical outcomes data from individual participants treated with the AAV gene therapy candidate.
FLT201 represents a potential breakthrough gene therapy approach for Gaucher disease, with the company advancing the candidate toward Phase 3 development.
Spur Therapeutics (search) announced it will present updated clinical data from its Phase 1/2 GALILEO-1 and GALILEO-2 trials of avigbagene parvec (search) (FLT201), a Phase 3 gene therapy candidate for Type 1 Gaucher disease (search), at the 22nd Annual WORLDSymposium taking place February 2-6, 2026 in San Diego.
Two-Year Follow-Up Data to Be Presented
The primary platform presentation, titled "Two-year follow up of FLT201 AAV gene therapy in adults with Type 1 Gaucher Disease (search): Results from GALILEO-1 and GALILEO-2," will be delivered by Dr. Ida Schwartz from Hospital de Clinicas do Porto Alegre, Brazil, during the Clinical Applications session on Thursday, February 5, at 9:30 a.m. PST.
The presentation will be accompanied by three poster sessions providing detailed analysis of the clinical outcomes. Dr. Ozlem Goker-Alpan from the Lysosomal and Rare Disorders Treatment Center in Virginia will present "Individual Participant Outcomes After FLT201 AAV Gene Therapy for Type 1 Gaucher Disease (search): Two-Year Biomarker and Clinical Data" during the Clinical Applications Poster Session on Thursday, February 5, at 3:30 p.m. PST.
Biomarker Analysis Focus
A third poster presentation by Simon Flynn from Spur Therapeutics (search) will examine "Lyso-Gb1 (search) Dynamics as a Surrogate Biomarker in Type 1 Gaucher Disease (search) Treated with FLT201 AAV Gene Therapy." This presentation will explore the potential of Lyso-Gb1 as a surrogate biomarker for monitoring treatment response in patients receiving the gene therapy.
Gene Therapy Development Program
FLT201 represents Spur Therapeutics (search)' lead gene therapy candidate utilizing adeno-associated virus (AAV) technology for Type 1 Gaucher disease (search). The company describes itself as a clinical-stage biotechnology company focused on developing gene therapies for debilitating chronic conditions by optimizing every component of its product candidates.
Beyond Gaucher disease, Spur Therapeutics (search) is also advancing a preclinical gene therapy candidate for Parkinson's disease (search) as part of its broader genetic medicine portfolio. The company states its mission is to "unlock the true potential of gene therapy to realize outsized clinical results."