Spur Therapeutics Reports Two-Year Follow-Up Data for AAV Gene Therapy FLT201 in Gaucher Disease

February 4, 2026-Spur Therapeutics has reported updated two-year follow-up data from its Phase 1/2 GALILEO-1 and GALILEO-2 studies evaluating avigbagene parvec (FLT201), an AAV-based gene therapy for Gaucher disease type 1. The results demonstrate sustained clinical benefit, robust biomarker improvements, and favorable safety following a single low-dose AAV infusion.

Data presented at the 22nd Annual WORLDSymposium™ showed continuous expression of the engineered enzyme GCase85 and rapid, durable reductions in lyso-Gb1, a validated biomarker of disease severity and a potential surrogate endpoint for accelerated approval. In patients with elevated lyso-Gb1 despite years of enzyme replacement therapy (ERT) or substrate reduction therapy (SRT), reductions ranged from 53% to 94% at two years.

All four patients treated at the low dose (4.5e11 vg/kg) discontinued prior ERT or SRT within 11 weeks of AAV gene therapy administration and remained off chronic therapy for up to 26 months. Improvements or stability were observed in hemoglobin levels, platelet counts, and liver and spleen volumes. Notably, new patient-reported outcomes showed improvements or maintenance of physical and mental well-being, with marked reductions in pain and fatigue in symptomatic patients.

FLT201 was generally well tolerated, with no dose-limiting toxicities reported. Spur Therapeutics has initiated a Phase 3 single-arm trial of AAV gene therapy FLT201, with FDA alignment to support potential accelerated and full approval pathways.