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 nNOS can improve blood flow to the muscle, thereby reducing muscle breakdown from ischemia and muscle fatigue, thus treating symptoms of DMD.
In addition to receiving an orphan drug designation from the EC, SGT-003 has also received an innovation license and access pathway (ILAP) designation in the UK. Furthermore, it has been granted a fast track, orphan drug, and rare pediatric disease designation by the U.S. Food and Drug Administration (FDA).
SGT-003 is currently being evaluated in a global first-in-human, open-label, single-dose, multi-centre Phase1/2 INSPIRE DUCHENNE trial on pediatric Duchenne's patients who have tested positive for mutations in the dystrophin gene. The Phase 3 IMPACT DUCHENNE trial is a simultaneously ongoing randomized, double-blind, placebo-controlled study for SGT-003 in individuals aged 7 to 12 years with a confirmed Duchenne's diagnosis.
SLDB is currently trading at $7.47, down 0.40%.
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