The study focuses on Gaucher Disease Type 1, a rare inherited condition in which the body lacks enough of the enzyme that breaks down a fatty substance called glucocerebroside. Without this enzyme, fat builds up in the spleen, liver, bones and blood, leading to anemia, low platelet counts, enlarged organs, and bone problems. The investigational treatment is a one‑time infusion of FLT201, a form of gene therapy that uses a harmless virus to deliver a working copy of the gene that makes the missing enzyme, beta-glucocerebrosidase. Participants may continue their usual medicines such as the oral solution containing sirolimus, the hard capsules with tacrolimus, the injectable form of methylprednisolone, and the tablet form of prednisolone while receiving the study drug.
The main aim of the trial is to see whether the single infusion of FLT201 can keep blood‑cell levels, spleen and liver size, and bone health stable over a year. After the infusion, participants attend regular clinic visits for about 12 months during which blood tests, imaging scans, and questionnaires are used to monitor safety and how well the therapy works. The study tracks changes in blood counts, organ volumes, enzyme activity, and quality‑of‑life measures without exposing participants to any unnecessary procedures.
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