 - Generally well-tolerated with a favorable safety profile in five patients - Serum beta-galactosidase enzyme activity increased in all patients at all timepoints between Day 7 and Month 6, representing an approximate doubling in enzyme activity after gene transfer - At Month 6, enzyme activity restored to 23-57 mean: 38 of normal reference levels - All five children demonstrated signs of clinical disease stability as assessed by Vineland-3 Growth Scale Value, Upright and Floor Mobility, and Clinical Global Impression CGI scales - High-dose cohort initiated in November 2020; two patients now dosed without complications 