Solid Biosciences said Monday that it has restarted a clinical trial involving its experimental gene therapy for Duchenne muscular dystrophy without the serious safety issues that forced the study to shut down in 2019.
The biotech also disclosed for the first time interim results from patients treated before the study was stopped, showing small improvements in muscle and lung function after one year of follow up.
The safe dosing of a newly enrolled patient represents an encouraging reset for Solid’s Duchenne gene therapy program, but it still lags far behind its two main rivals, Sarepta Therapeutics and Pfizer. And optimism for all of these programs has cooled considerably ever since Sarepta announced in January disappointing results from its first randomized clinical trial.
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