India, Feb. 18 -- Solid Biosciences Inc. (SLDB) Tuesday reported positive initial data from the Phase 1/2 INSPIRE DUCHENNE study of its gene therapy product candidate SGT-003 for the treatment of Duchenne muscular dystrophy. Following the news, the company's shares were more than 75 percent up in pre-market.
In the study, SGT-003 was administered as a one-time intravenous infusion in 6 participants, as of February 11, 2025. Interim data reported in the first three participants showed an average microdystrophin expression of 110%. Microdystrophin expression is a measure of the production of a shortened, functional gene that's used to treat Duchenne. Significant improvements in multiple additional muscle health biomarkers were also observed....