What is ST-920 and how does it work?
In Fabry disease, the gene called galactosidase alpha (GLA), that provides instructions for making an enzyme called alpha-galactosidase A (α-Gal A), does not function correctly.
When α-Gal A enzyme is produced in low quantities, a fatty substance called Gb3 builds up in the tissues, causing disease in vital organs.
ST-920 is designed to deliver a healthy copy of the GLA gene to the liver. The liver should then be able to produce the α-Gal A enzyme and secrete it via the blood stream to the rest of the body. It is hoped that a potential increase in the α-Gal A enzyme in the blood could reduce the need for use of current treatments for Fabry disease.
More information on Fabry disease