About SOLID BIOSCIENCES INC
Solid Biosciences Inc. is a life science company. The Company is focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Its lead program, SGT-001, is a gene transfer candidate under investigation for its ability to drive functional dystrophin protein expression in patient’s muscles and improve the course of the disease. SGT-001 is designed to address the underlying genetic cause of Duchenne by delivering a synthetic transgene that produces dystrophin-like protein that is only expressed in muscles of the body, including cardiac and respiratory muscles. Its SGT-001 vector is derived from a non-pathogenic virus called adeno-associated virus (AAV), which was selected for its ability to enter skeletal, diaphragm and cardiac muscle tissues. The vector is designed to carry a synthetic dystrophin transgene construct, called microdystrophin, that retains the components of the dystrophin gene to fit within AAV packaging constraints.