A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease.

Fabry Disease (X-linked lysosomal storage disease).

1. Incidence of treatment-emergent adverse events (TEAEs). Additional safety evaluations will include: o Routine hematology, chemistry, and liver tests, vital signs, electrocardiogram (ECG) and echocardiogram (ECHO). o Serial alpha fetoprotein (AFP) testing and magnetic resonance imaging (MRI) of liver (or equivalent imaging modality) to monitor for liver mass.

1. Change from baseline at specific time points over the 1-year study period in: o α-Gal A activity in plasma o Gb3 and lyso-Gb3 levels in plasma, 2. Change from baseline at specific time points over the 1-year study period in: o Frequency of ERT infusion, 3. Change from baseline at specific time points over the 1-year study period in: o Estimated glomerular filtration rate (eGFR) using the CKD-EPI formula, 4. Change from baseline at specific time points over the 1-year study period in: o Ejection fraction, global longitudinal strain, left ventricular mass index (LVMI), left ventricular systolic function measured by cardiac magnetic resonance imaging (CMR), 5. ST-920 vector clearance measured by level of vector genome in blood (plasma), saliva, urine, stool, and semen (if applicable)

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Germany, Italy