Challenges in Conducting Research in DMD


The co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed some challenges the company is facing.

“We've always known that clinical endpoints were going to be challenging in Duchenne... [knowing that], we designed a study that takes almost every possible endpoint into consideration. It's very exploratory. It has a number of primary and secondary end points and it is always looking at both biomarkers and clinical benefits.”

Ilan Ganot, co-founder, president, and chief executive officer of Solid Biosciences, has a personal reason for finding an effective, targeted treatment for Duchenne muscular dystrophy (DMD): his son has been diagnosed with the disease. His company is using advanced gene therapy approaches to try and fill a great unmet need for patients with the neuromuscular disease.

SGT-001, their lead program and a gene therapy transfer agent, is currently in phase 1/2 clinical trials. It has continued to show safety and efficacy in the first 8 patients dosed, and data from the first 6 patients showed that those who received a high-dose version exhibited 5% to 17.5% of normal microdystrophin expression levels by Western blot and 20% to 70% of positive muscle fibers by immunofluorescence.

In an interview with GeneTherapyLive, Ganot discussed the challenges of conducting research and studies in DMD. He also shared how a big part of the company’s focus right now is on solving manufacturing challenges for SGT-001 and future products in their pipeline.

Morris CA, Clary CM, Redican S, et al. IGNITE-DMD phase I/II study of SGT-001 microdystrophin gene therapy for Duchenne muscular dystrophy. Presented at: 2021 American Society of Gene & Cell Therapy Annual Meeting; May 12-15, 2021. Abstract 263
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