To support the advancement of its clinical program, Solid has opened an office in London, England. Spearheaded by leading DMD clinician Valeria Ricotti, M.D., Ph.D., (formerly University College London, Institute of Child Health and Great Ormond Street Hospital) and patient advocate Kerry Rosenfeld, Solid’s London team will work closely with the European DMD community to help realize its goal of bringing meaningful therapies to patients around the world.
“The orphan designations mark a positive step forward in our efforts to advance SGT-001 through development and to patients with DMD in both the United States and Europe,” said Ilan Ganot, chief executive officer and founder of Solid Biosciences. “Our teams in the United States and in our new offices in London are working tirelessly to bring our gene therapy candidate SGT-001 to patients as soon as possible.”
Duchenne muscular dystrophy is a rare, progressive muscle-wasting disease that affects approximately 1 in 3,500-5,000 boys born worldwide and is the most common fatal genetic disorder diagnosed in childhood. It is caused by the absence of the dystrophin protein, the fundamental mediator of skeletal and cardiac muscle function1. SGT-001 is an adeno-associated viral (AAV) vector-mediated gene therapy candidate that is being developed for its potential to restore functional dystrophin expression in muscle.
In preclinical studies, a single administration of SGT-001 resulted in long-term, systemic expression of micro-dystrophin, a shorter form of dystrophin, in muscle. SGT-001 also improved function, as measured by improved muscle strength and protection against contraction-induced damage.
Orphan Drug designations offer companies incentives and support for developing therapies for rare diseases. In the U.S., the FDA's Office of Orphan Products Development grants Orphan Drug Designation to therapies intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States2. In the European Union, the European Medicines Agency (EMA) renders an opinion on Orphan designation applications for candidates intended for the treatment of life-threatening or chronically debilitating rare conditions with a prevalence of less than five in 10,000 in the European Union.3 The European Commission then adopts the EMA’s opinion through a separate approval process.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare, muscle-wasting disease. As the most common fatal genetic disorder diagnosed in childhood, it affects approximately 1 in 3,500-5,000 boys born worldwide, yet there is a significant need for treatments that can benefit all boys with the disease. DMD is caused by the absence of dystrophin, a protein that is fundamental for muscle function. Because of the lack of dystrophin, patients experience progressive and pervasive muscular degeneration, which eventually results in premature death. Patients are typically wheelchair-bound by their early teens and succumb to respiratory or heart failure in their 30s.