Fabry disease is a metabolic disease characterized by a deficiency in the lysosomal α-galactosidase enzyme caused by mutations in the GLA gene. This leads to substrate accumulation in the lysosomes, cellular dysfunction and organ damage.

At the recent ASGCT meeting, researchers from Exegenesis Bio Inc. presented preclinical data for EXG-102-031, a novel recombinant adeno-associated virus (rAAV)-gene therapy being developed for the treatment of neovascular age-related macular degeneration (AMD), also called wet AMD (wAMD).