India, April 28 -- Solid Biosciences Inc. (SLDB), a life sciences company, on Tuesday announced receipt of an Orphan Drug Designation from the European Commission for SGT-003 in treating Duchenne's muscular dystrophy.
Duchenne's muscular dystrophy (DMD) is a rare, muscle-wasting genetic disorder that is irreversible, progressive, and ultimately fatal. As an X-linked genetic disorder, it largely affects males and has an estimated prevalence of about 10,000-15,000 cases in the U.S. alone.
SGT-003 is a novel, investigational gene therapy containing a microdystrophin construct and a next-generation capsid, POLARIS-101. The microdystrophin construct includes R16/17 domains, which localize nNOS to the muscle. Nonclinical studies have shown that...