During the study, participants received a low dose of 4.5 hundred billion vector genomes per kilogram of body weight (vg/kg) of FLT201 and were closely monitored for clinical outcomes.

Pamela Foulds, MD, the chief medical officer of Spur Therapeutics, highlighted the therapy's potential to establish new standards of care for serious diseases.

Notably, four participants were able to discontinue enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) within three months of treatment, maintaining this status for up to 21 months.

Participants exhibited stable hemoglobin levels and either stable or improved platelet counts for up to 18 months post-treatment, indicating strong safety and efficacy signals.

The experimental gene therapy FLT201 has demonstrated sustained clinical benefits for up to 21 months in patients with Gaucher disease type 1, as revealed by data from the Phase 1/2 GALILEO study.

The GALILEO-1 study involved six adults with Gaucher type 1, all of whom had been on standard treatments for at least two years prior to receiving FLT201.

Data also showed significant reductions in glucosylsphingosine (lyso-Gb1), a critical biomarker of disease burden, sustained for up to 15 months after stopping standard therapies.

While some patients developed temporary antibodies against the enzyme following treatment, this did not compromise the overall efficacy of the therapy.

Animal model data presented alongside the human study indicated stable enzyme levels for over 3.5 years following a single dose of FLT201.

These promising findings were presented at the 28th annual meeting of the American Society of Gene and Cell Therapy (ASGCT) held in New Orleans.