Fabry Genetic Medicines

Fabry Genetic Medicines refers to a discovery-stage gene therapy program being developed by Amicus Therapeutics for the treatment of Fabry disease. Fabry disease is a rare, X-linked lysosomal storage disorder caused by mutations in the GLA gene, which leads to a deficiency of the alpha-galactosidase A (α-Gal A) enzyme and the subsequent systemic accumulation of globotriaosylceramide (Gb3). This genetic medicine approach aims to deliver a functional version of the GLA gene to patient cells, thereby restoring endogenous production of the α-Gal A enzyme. This program represents a next-generation effort by Amicus to provide a potentially curative, one-time treatment for Fabry disease, expanding upon their existing commercial portfolio for the condition, which includes the pharmacological chaperone Galafold (migalastat).

Other names

Amicus Fabry gene therapy