Report Summary

Assessment of Toxicity and Biodistribution of the AAV2/8LSPhGAApA

Study Summary

Overall Study Design 
The overall purpose of the gene therapy product AAV28LSPhGAApA adeno-associated vector liver-specific promoter 
human acid alpha glucosidase gene with poly adenosine is to modulate the immune response to recombinant human acid 
alpha glucosidase rhGAA given as enzyme replacement therapy ERT in patients with infantile-onset glycogen storage 
disease type II GSD-II Pompe disease and with no residual GAA protein in their bodies 
The goals of the current study were to 1 assess toxicity following administration of the gene therapy product in a manner 
similar to the intended clinic use in GAA-knockout mice an animal model of the Pompe disease 2 determine biodistribution 
of the gene therapy product and 3 confirm the efficacy of the gene therapy product as an adjunctive therapy to rhGAA 
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