by: Parent Project Muscular Dystrophy
Solid Biosciences recently joined PPMD for a community webinar to provide an overview of INSPIRE DUCHENNE, a first-in-human clinical study evaluating SGT-003, a next-generation microdystrophin gene therapy candidate for the treatment of Duchenne.
The Solid team touched on the company’s previous clinical experience using a first generation AAV candidate, SGT-001. The insights they gained from the IGNITE DMD study of SGT-001 advanced their next generation microdystrophin gene therapy development. Solid indicated that they are now developing a potentially more muscle-targeted product with an optimized purity profile to achieve similar or improved efficacy at lower dose levels as compared to first generation gene therapy products.
In their initial research with mice, Solid has found that SGT-003 microdystrophin may provide additional protection from muscle damage. The company is now working on the clinical site startup for INSPIRE DUCHENNE at Nationwide Children’s Hospital in Columbus, Ohio, and at UCLA in Los Angeles, California. The Solid team covered both key inclusion and exclusion criteria for the study, as well as shared details about the key study assessments. Finally, the Solid team answered questions from the community on a variety of topics.
