The U.S. Food and Drug Administration (FDA) has awarded Rare Pediatric Disease Designation for SGT-003, a gene therapy designed for Duchenne Muscular Dystrophy (DMD) treatment.
Duchenne Muscular Dystrophy (DMD) is a genetic disorder that leads to progressive muscle degeneration and weakness. The disease is caused by genetic mutations in the DMD gene. Common symptoms include atrophy of skeletal and heart muscle, muscle weakness, delayed ability to sit, stand, or walk, and difficulties learning to speak.
SGT-003
SGT-003 an adeno-associated virus (AAV) gene therapy that targets the dystrophin gene causing DMD symptoms. The therapy involves delivery of shortened dystrophin protein and nNOS binding domain, both essential in restoring muscle function. INSPIRE Duchenne is a phase 1/2, first-in-human, open-label, multicenter trial planned to determine SGT-003 safety and tolerability in pediatric patients. The first updates from this trial are expected mid-2024.
The Rare Pediatric Disease Designation received by SGT-003 encourages the development of new treatment options for rare diseases by granting a Priority Review Voucher (PRV) upon approval of the treatment.
For more information on DMD and other rare musculoskeletal disorders, visit https://checkrare.com/diseases/musculoskeletal-diseases/