Solid Biosciences CEO Bo Cumo discusses recent DMD data for the company's microdystrophin gene therapy, and explains the development and regulatory steps ahead

Solid Biosciences' CEO, Bo Cumbo, discusses the company's gene therapy advancements for Duchenne muscular dystrophy, emphasizing their focus on improved delivery methods via the Polaris capsid. Cumbo highlights recent data from a Phase 1-2 study involving 41 patients, noting the comprehensive safety profile and consistent microdystrophin expression across various assays. The discussion covers the durability of the therapy, with positive results seen in patients followed for a year, and the potential for cardiac benefits. Cumbo also addresses the FDA's review process for accelerated approval, including upcoming meetings to align on functional data and surrogate markers. He also touches on Solid's Friedreich's ataxia program and its potential to transform the treatment landscape.