Addressing Challenges and Opportunities in Treating Duchenne Muscular Dystrophy


The co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed promising updates from their phase 1/2 IGNITE-DMD trial.

"Duchenne muscular dystrophy is a really challenging disease to measure in the clinic. It's easy to measure the biomarker; it's more difficult to correlate that with outcomes. But from what we have seen, which is just very few patients at around a 1-year time point, we are certainly encouraged and excited that we have a treatment that may well be improving outcomes.”

Finding an effective, targeted treatment for Duchenne muscular dystrophy (DMD) is personal for Solid Biosciences co-founder Ilan Ganot, whose son has been diagnosed with the disease. The company, for which Ganot also serves as president and chief executive officer, is harnessing advanced gene therapy approaches to try and fill a great unmet need for patients with the neuromuscular disease.

Solid considers their approach to research and development “technology agnostic,” in that they are more focused on the treatment strategies that will solve the most problems for patients versus those that can be addressed by a single approach. Their lead program, a gene therapy transfer agent, is currently in phase 1/2 clinical trials.

SGT-001 has continued to show safety and efficacy in the first 8 patients dosed. 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 shared more about the company’s motivations and how Solid is persevering in DMD despite inherent clinical challenges.  

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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