EXG110

EXG110 is a one-time adeno-associated virus (AAV)-based gene therapy developed for the treatment of Fabry disease. It is designed to deliver a functional copy of the GLA gene directly to liver and heart cells, enabling production of the alpha-galactosidase A enzyme. This approach aims to clear toxic sphingolipid buildup in tissues caused by deficient alpha-galactosidase A activity in Fabry disease patients. EXG110 uses a proprietary capsid for enhanced delivery efficiency and safety, potentially allowing lower dosing and improved outcomes compared to existing therapies. The drug has received orphan drug designation from the US FDA and is currently being evaluated in clinical trials[1][2][3][5].