FACTs Fabry gene therapy clinical trial: Two-year data

Fabry disease is due to a deficiency in alpha-galactosidase A (a-gal A) activity. Enzyme therapy is used to treat Fabry disease although the cost is high and biweekly infusions are required long-term. We have shown that when a-gal A is overexpressed in cells, a portion of the hydrolase is secreted and can be taken up and used functionally by unmodified bystander cells. Based on this concept, and building on extensive preclinical data we …