Solid Bio is hit by another setback as initial biopsies spotlight a flop for Duchenne MD
Solid Biosciences has had another setback.
The biotech announced this morning that its dose-ascending Phase I/II study of its lead gene therapy for Duchenne muscular dystrophy flopped, sending the company into overdrive to hustle a higher dose into the clinic.
The company announced Thursday morning that a 3-month biopsy of a handful of patients “showed low levels of microdystrophin protein expression.” In 2 of 3 patients, protein expression was undetectable by western blot analysis. In the third patient the dystrophin expression hit only 5% by western blot analysis, while analysts and the company were looking for 10% or better.
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