This clinical trial is focused on studying Gaucher Disease Type 1, a genetic disorder that affects the body’s ability to break down a certain type of fat, leading to its buildup in various organs. The study is investigating a treatment called FLT201, which is a special type of therapy using an adeno-associated viral vector. This vector is designed to deliver a modified gene that helps produce an enzyme called beta-glucocerebrosidase, which is deficient in people with Gaucher Disease. The treatment is given as a solution for infusion, meaning it is administered directly into the bloodstream.

The purpose of this study is to assess the long-term safety of FLT201 in individuals who have Gaucher Disease Type 1. Participants in the study will be monitored over an extended period to observe how their bodies respond to the treatment. This includes checking for any side effects and measuring changes in various health indicators, such as enzyme activity levels and organ sizes, using methods like MRI scans. The study will also track the presence of the treatment in the body over time.

Participants in this study are those who have previously received the FLT201 treatment. They will be required to attend regular follow-up visits to ensure their safety and to gather important data on the treatment’s effects. The study aims to provide valuable information on the safety and effectiveness of this innovative therapy for managing Gaucher Disease Type 1.