On October 1, 2020, Solid Biosciences announced that the FDA (U.S. Food and Drug Administration) has lifted the clinical hold placed on the company’s IGNITE DMD Phase I/II clinical trial, investigating SGT-001, a novel gene therapy for Duchenne muscular dystrophy.
This trial was put on hold by the FDA last year due to a serious adverse event. Solid has successfully addressed all the FDA’s questions, provided updated safety and efficacy data for all patients dosed in this study. There have been no additional drug-related adverse events up to 30 months post-dosing. They will begin screening patients as soon as possible with anticipated dosing in the first quarter of 2021. They have also made several changes to their manufacturing and study procedures, including the implementation of clinical risk mitigation strategies to enhance patient safety.
What is gene therapy?
Gene therapy is an important therapeutic approach and works by correcting the defected DNA by delivering a functional piece of DNA or RNA into the muscle cells and restoring the function of that cell. To do this it uses a non-disease carrying virus-like AAV in the case of Solid’s approach, with a mini-dystrophin gene (DNA) and other components to make the functional dystrophin protein.
Solid has 1 of 3 micro or mini dystrophins currently in Clinical trial. Sarepta Therapeutics is working on their SRT-9001 and Pfizer are working on their PF-06939926.
About Solid Biosciences
Solid Biosciences is a life science company singularly focused on developing meaningful therapies for Duchenne muscular dystrophy (Duchenne). We aim to be the center of excellence across the entire disease spectrum, bringing together those with expertise in science, technology, disease management and care. Disease-focused and founded by those directly impacted by Duchenne, our mandate is simple yet comprehensive – attack all facets of the disease and improve the daily lives of patients living with this devastating disease