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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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.
Recent adverse events are ‘speed bumps’ that need to be worked around, biologics center head Peter Marks says; Morgan Stanley’s Peter Hwang asserts high-profile safety issues have added to unpredictability of gene therapy development, dampening investor enthusiasm for the field.