Solid Restarts DMD Gene Therapy Trial, With Encouraging Interim Data To Back It Up
The Phase I/II trial testing SGT-001 in patients with Duchenne muscular dystrophy resumed after FDA lifted a clinical hold on the trial; interim data from the first six patients were encouraging.
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Sio Gene, Genocea, Solid Biosciences, Saniona and Black Diamond join First Wave, Zosano, Vallon, Finch, Magenta, ProQR and Imara on the growing list of biopharma firms reducing headcount to make their cash last until valuations return to a level that may support new fundraising.
The company has initiated the first pivotal double-blind gene therapy trial in DMD in the US in a tight race with Pfizer.
Advisory committee will discuss five types of toxicity risks with adeno-associated virus vector-based gene therapy products at a two-day meeting in September, which follows some recent high-profile instances of adverse events that have delayed development programs.