AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

Inclusion Criteria: * Diagnosis of Pompe disease by blood or skin fibroblast GAA assay and two pathogenic variants in the GAA gene, * Age: Greater than or equal to 18 years at enrollment. * Subjects are capable of giving written informed consent. * Able to walk at least 100 meters on the 6MWT (with assistive devices permitted). * FVC within the range of 30% to less 90% (inclusive) of predicted in the upright position. * Subjects with a confirmed diagnosis of LOPD who have been treated with ERT for at least 104 weeks (inclusive) immediately preceding screening and receiving a stable dose of ERT for the 52-week period immediately preceding dosing. Exclusion Criteria: * Invasive ventilation required or noninvasive ventilation required while awake and upright. * FVC \<20% of predicted (supine). * Received any live vaccination 2 months prior to study Day 1. * Pregnant or nursing mothers. * Serology consistent with exposure to HIV, or serology consistent with active hepatitis A, B or C infection. Any active liver disease. * Active infection based upon clinical symptoms. * Having started respiratory muscle strength training in the last 6 months prior to study day 1 or having discontinued respiratory muscle strength training in the 6-month period preceding study day 1, or having started respiratory strength training greater than 6 months prior to study day 1 and unwilling to continue for the first year of study participation. * Received an investigational drug or participated in a…