Solid Biosciences announced a new strategic collaboration with Ultragenyx, a biopharmaceutical company with a successful track record of bringing novel therapies to patients with serious rare and ultra-rare genetic diseases. This new partnership is great news for our community as it will advance meaningful treatment options for Duchenne muscular dystrophy.
Solid remains committed to its current gene therapy program and expects to start its next patient in the IGNITE DMD trial in the first quarter of 2021.
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 to make a functional protein. 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