SGT-701

SGT-701 is an early-stage gene therapy candidate being developed by Solid Biosciences for the treatment of genetic cardiac diseases, specifically BAG3-mediated dilated cardiomyopathy. The program was acquired through Solid's 2022 acquisition of AavantiBio and is supported by exclusive licensing agreements with the University of Florida Research Foundation (UFRF). SGT-701 utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the BCL2-associated athanogene 3 (BAG3) gene to the myocardium. BAG3 plays a vital role in cardiac muscle cells by maintaining the structural integrity of the sarcomere and facilitating the clearance of misfolded proteins; mutations in this gene are a known cause of familial dilated cardiomyopathy. SGT-701 is currently in preclinical development, aiming to provide a precision medicine approach to heart failure by addressing the underlying genetic deficiency.