4D-310 gene therapy granted FDA Fast Track Designation for treatment of Fabry Disease

Fabry disease is a debilitating lysosomal storage disorder caused by a deficiency of alpha-galactosidase A (AGA) enzyme activity. 4D-310 is designed to drive high level AGA enzyme expression within the diseased target tissues, as well as high and stable blood concentrations.

Source:

Biospace Inc.