AVB-401

AVB-401 is a preclinical gene therapy candidate developed for the treatment of BAG3-mediated dilated cardiomyopathy (DCM). The therapy aims to address genetic insufficiency of the BAG3 protein in cardiac muscle by delivering a functional copy of the BAG3 gene using an adeno-associated virus (AAV) vector. The construct utilizes a cardiac-specific promoter and AAV capsid designed to enhance expression in heart tissue while minimizing off-target effects in other organs such as the liver. Originally developed by AavantiBio and now under Solid Biosciences following acquisition, AVB-401 is part of a pipeline focused on precision genetic medicines for rare cardiovascular diseases[1][2][4][5][9].

Other names

AVB 401AVB401AVB-401AVB-401 BAG3 DCMAVB401 BAG3 DCMAVB 401 BAG3 DCMBAG3-DCMBAG-3-DCMBAG 3-DCM