Unique Challenges With Gene Therapy in Duchenne Muscular Dystrophy

“I would say that my philosophy has always been that our job at Solid is to derisk the approach and derisk [Duchenne muscular dystrophy] and continue to innovate and push the envelope as far as science can go. Six, 7 years ago, treating Duchenne patients with gene therapy was simply crazy. Now, it's obvious and everybody's doing it.”

Solid Biosciences is using advanced gene therapy approaches to try and fill a great unmet need for patients with Duchenne muscular dystrophy (DMD). The mission is personal for the company’s co-founder, president, and chief executive officer, Ilan Ganot: his son has been diagnosed with the disease.

Their lead program, SGT-001, 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.

GeneTherapyLive spoke with Ganot about the challenges in using gene therapy in DMD, such as the transduction of the therapy to the muscles. He discussed the continual promising data in SGT-001 as well as another program in development, SGT-003, which is focused on identifying and utilizing a more efficient vector for gene therapy.

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