On May 13, 2020, Solid Biosciences announced an update on their phase 1/2 clinical trial on SGT-001, an investigational gene therapy under development for Duchenne.
This trial is currently on hold due to some safety concerns, but patients enrolled are continuing to engage in study-related activities. Their clinical biomarker results presented at a recent scientific meeting continues to support their development and we hope to hear more from them on their next steps.
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 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