SGT-601

SGT-601 is an adeno-associated virus (AAV) gene therapy candidate designed for the treatment of TNNT2-mediated dilated cardiomyopathy (DCM). Developed by Solid Biosciences following its acquisition of AavantiBio, the therapy utilizes a novel, rationally engineered muscle-tropic capsid, AAV-SLB101, to deliver a genetic payload consisting of the human wild-type cardiac troponin T (cTnT) cDNA. This payload is driven by a cardiac-specific promoter to ensure targeted expression in the heart. SGT-601 aims to address the underlying cause of DCM in patients with TNNT2 pathogenic variants, which typically lead to calcium desensitization and impaired myocardial contractility. Preclinical data presented at ASGCT 2026 demonstrated that systemic administration of SGT-601 improved left ventricular ejection fraction, reduced ventricular volumes, and increased survival in R141W knock-in mouse models, while also showing a favorable safety profile in non-human primates.