New DMD Drug Candidate Designated Orphan Drug

Clinical studies for the drug candidate are anticipated to begin in 2017.
Clinical studies for the drug candidate are anticipated to begin in 2017.

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.

For more information call (617) 337-4682 or visit SolidBio.com.