Empowered Patient Podcast

Dr. Gabriel Brooks is Chief Medical Officer at Solid Biosciences, a precision molecular genetic medicines company focused on rare cardiovascular and neuromuscular diseases, including Friedreich's ataxia. Currently, there are very limited treatments for this rare, progressive neurologic disease caused by a genetic deficiency. Solid Biosciences' novel gene therapy uses dual-route administration to deliver directly to the heart and brain and to replace the missing frataxin gene, which is critical for energy production. Dr. Brooks explains, "Our flagship program is our DMD program, where we have two clinical trials, a first-in-human INSPIRE study, and a double-blind randomized placebo-controlled phase three trial for the SGT-003 DMD medicine. And for Friedreich's ataxia, we have the SGT-212 program, which uses a novel dual route of administration to target not only the cardiomyopathy, but also uses direct injection into the dentate nucleus. We're trying to address the central pathophysiology of the ataxia that pa