Roche Pharmaceutical Development and Sales Overview

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 Safety Primary endpoint FPI Q4 2020 Status Recruitment completed Q2 2022 CT Identifier NCT04093349 Spark THERAPEUTICS TM 143 Roche Metabolic Diseases

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