Solid Biosciences and Solid GT announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation for SGT-001 for the treatment of patients with Duchenne muscular dystrophy (DMD). 

DMD, a rare, progressive muscle-wasting disease is the most common fatal genetic disorder diagnosed in childhood. SGT-001 is an investigational adeno-associated viral (AAV) vector-mediated gene therapy that works by potentially restoring functional dystrophin expression in muscle.  

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Data from preclinical studies showed that a single administration of SGT-001 led to long-term, systemic expression of micro-dystrophin, (a shorter form of dystrophin) in muscle. Use of SGT-001 also improved function, as measured by improved muscle strength and protection against contraction-induced damage.

SGT-001 is being studied in IND-enabling studies and clinical studies for the drug candidate are anticipated to begin in 2017.

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