Half-Year 2022 Financial and Clinical Trials Update

Pompe disease Unique gene therapy platform Molecule SPK-3006 (RG6359) Indication Phase/study # of patients ▪ Gene transfer study for late-onset Pompe disease Pompe disease Phase I/II RESOLUTE N=20 Design Primary endpoint Status CT Identifier Safety FPI Q4 2020 NCT04093349 Spark THERAPEUTICS TM 162 Roche Metabolic Diseases

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