A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Exclusion Criteria

• •Active hepatitis B and/or C;
• •Significant underlying liver disease;
• •Human immunodeficiency virus (HIV) infection;
• •Prior hypersensitivity to rhGAA;
• •Pre-existing anti-AAV neutralizing antibody titers;
• •High titer antibody responses to rhGAA;
• •Requires any invasive ventilation or requires noninvasive ventilation while awake and upright;
• •Received any prior vector or gene transfer agent;
• •Active malignancy (except non-melanoma skin cancer);
• •History of liver cancer;
• •Pregnant or nursing women;
• •Any evidence of active infection at the time of SPK-3006 infusion.