The therapy has demonstrated a favorable safety profile in clinical evaluation (PMID 39794302). Across the study, 228 adverse events (AEs) were reported, the majority of which were mild to moderate. Only 20 AEs were classified as Grade 3–4 (more serious). Importantly, no severe adverse events (SAEs) were determined to be related to the cell therapy product. Key organ function measures, including cardiac and renal parameters, remained stable over five years of follow-up. This is particularly important given these are the organ systems typically affected by the disease. Following treatment under an outpatient conditioning protocol, patients experienced an initial rise in enzyme activity. As expected in cell-based therapies, this early peak gradually declined and stabilized at a sustained level over time. A similar pattern was observed in vector copy number (VCN), a measure of how many therapeutic gene copies are present in treated cells (data not shown). VCN initially peaked at approximately 1 copy per cell and then stabilized at around 0.2 copies per cell by the second year after treatment. This stabilized level remained durable over long-term follow-up. Based on these observations, repeat dosing may provide additional benefit for some patients. A second treatment could potentially increase the stabilized VCN baseline, which may enhance long-term enzyme production and therapeutic effect.