SGT-501

SGT-501 is an investigational gene therapy being developed by Solid Biosciences for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), specifically CPVT type 1 (CPVT-1), which is caused by a gain-of-function mutation in the ryanodine receptor 2 (RYR2) gene. The therapy aims to address abnormal cardiac calcium handling, which underlies arrhythmias in CPVT, by modulating the function or expression of key proteins involved in cardiac excitation-contraction coupling, notably RYR2 and calsequestrin 2 (CASQ2). SGT-501 utilizes a gene therapy modality and is currently undergoing IND-enabling GLP toxicology studies. The company anticipates submitting an Investigational New Drug (IND) application to the FDA in the first half of 2025. The FDA has granted Rare Pediatric Disease designation to SGT-501[6][8][9][5].