Reena Sharma, MD, on Assessing Spur Therapeutics’ Gene Therapy FLT201 in Gaucher Disease

“It certainly offers a very promising result and makes a case for exploring further as a treatment for this group of patients in the future, which can not only stabilize the condition, but also actually take away some of the disease burden that has not been managed by the current therapy.”

Currently, standard of care (SOC) treatment options for Gaucher disease include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). Although these options can provide a meaningful clinical benefit, they have a substantial burden of care, as they must be regularly administered to patients throughout their lifetime. In order to address this unmet need, Spur Therapeutics is currently developing FLT201, an adeno-associated virus (AAV) vector-based gene therapy, with the intention of providing a one-time treatment option for Gaucher disease. The therapy is currently being evaluated in the phase 1/2 GALILEO-1 clinical trial (NCT05324943). Interim results from this study were recently presented in a poster at the 21st Annual WORLDSymposium, held February 3 to 7, 2025, in San Diego, California.¹

During the conference, CGTLive® reached out to Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, who presented the data at WORLDSymposium, to learn more. Sharma explained what FLT201 is and how it functions, and then went over the key results that were presented and their implications. She noted that the gene therapy uses an AAVS3 capsid designed for efficient liver uptake and delivers a transgene encoding an engineered enzyme (GCase85), which is designed to be longer-acting than the natural GCase enzyme that is found at deficient levels in patients with Gaucher disease.² Sharma highlighted that all 5 patients included in the efficacy analysis presented showed expression of GCase85 and that 4 of these 5 patients were able to come off of their SOC therapy within 4 to 11 weeks of treatment with FLT201.

REFERENCES
1. Sharma R, Goker-Alpan O, Schwartz I, Giraldo P, Ferrante F, Foulds P. Results from GALILEO-1, a first-in-human clinical trial of FLT201 AAV gene therapy in adult patients with Gaucher Disease Type 1. Presented at: WORLDSymposium, held February 3-7, 2025, in San Diego, California. Poster #318
2. Spur Therapeutics announces positive data from phase 1/2 GALILEO-1 trial of FLT201, its gene therapy candidate for Gaucher disease, at WORLDSymposium. News release. Spur Therapeutics. February 4, 2025. Accessed February 4, 2025. https://www.globenewswire.com/news-release/2025/02/04/3020229/0/en/Spur-Therapeutics-Announces-Positive-Data-from-Phase-1-2-GALILEO-1-Trial-of-FLT201-Its-Gene-Therapy-Candidate-for-Gaucher-Disease-at-WORLDSymposium.html