AVR-RD-01 is an autologous drug product comprising a CD34+ cell-enriched fraction that contains cells transduced with a lentiviral (LV) vector encoding human GLA. A total of 20 classical FD male patients (≥16 years old), who had not previously received ERT and/or chaperone therapy within 3 years of the time of screening, received a one-time intravenous administration of AVR-RD-01 and were observed for 64 weeks. The gene discussed is CD34; the disease is Fabry disease.