Solid Biosciences is engaged in the treatment of Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys, with symptoms that manifest between three and five years of age. The company's primary gene transfer candidate, SGT-001 is designed to address the underlying genetic cause of DMD by delivering a synthetic transgene that produces dystrophin-like protein that is only expressed in muscles of the body, including cardiac and respiratory muscles. The company's portfolio also includes an initial disease modifying candidate, Anti-LTBP4, a monoclonal antibody designed to reduce fibrosis and inflammation by targeting and stabilizing the LTBP4 protein.
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