AT003

AT003 is an adeno-associated virus (AAV) gene therapy being developed for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT) caused by mutations in the calsequestrin 2 (CASQ2) gene. CPVT is a rare genetic condition that leads to life-threatening arrhythmias triggered by physical activity or emotional stress. AT003 is designed to deliver a functional version of the CASQ2 gene to cardiac muscle cells, aiming to restore normal calcium regulation and prevent the onset of arrhythmias. The program was originally initiated by Cardiogen Sciences and was subsequently acquired by Audentes Therapeutics, which is now a subsidiary of Astellas Pharma.