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Solid Biosciences stock price drops after gene therapy trial data release
Investing.com – Solid Biosciences Inc. stock dropped more than 8% on Wednesday after the company announced data from its Duchenne muscular dystrophy gene therapy trial. The stock was briefly halted earlier in the day.
The company released interim results from its Phase 1/2 INSPIRE DUCHENNE trial, which is testing SGT-003, a gene therapy for Duchenne muscular dystrophy. As of Wednesday, 40 participants had received treatment.
The trial showed restoration of microdystrophin protein expression and components of the dystrophin-associated protein complex. The company also reported improvements in muscle integrity markers measured by left ventricular ejection fraction, with cardiac function stabilizing.
Muscle biopsy results from 20 participants at day 90 showed an average microdystrophin protein expression level of 60% via Western blot analysis. Three participants at day 360 showed an expression level of 91%. β- dystroglycan positive fibers recovered by 60% at day 90 and 69% at day 360.
The company reported a 38% reduction in serum creatine kinase, and a 44% reduction in embryonic myosin heavy chain positive fibers at day 90. Solid Biosciences stated these results indicate the disruption of the chronic degeneration-regeneration cycle characteristic of Duchenne muscular dystrophy.