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Solid Restarts DMD Gene Therapy Trial, With Encouraging Interim Data To Back It Up

 
• By Jessica Merrill

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.

Railroad tracks seen from the back of an Adirondack train heading to Montreal, Canada, from New York, USA.
Solid's DMD gene therapy is back on track • Source: Shutterstock

Solid Biosciences Inc. has resumed the Phase I/II IGNITE DMD trial of the gene therapy SGT-001, dosing a seventh patient with Duchenne muscular dystrophy in the study. Meanwhile, the company also announced on 15 March encouraging interim data from the first six patients dosed in the study, improving the outlook for the gene therapy, though not eliminating uncertainty in what is a challenging area of drug development.

Solid is one of several companies in the early race to bring a gene therapy to market for DMD, but has faced several setbacks, including two clinical trial holds. The...

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