Challenges in Conducting Research in DMD

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.

REFERENCE

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