Solid Biosciences Receives FDA Rare Pediatric Disease Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia

Core Insights - Solid Biosciences has received FDA Rare Pediatric Disease and Fast Track designations for SGT-212, a gene therapy for Friedreich's ataxia [1][3] - SGT-212 is the only dual route gene therapy in development for Friedreich's ataxia, utilizing both intradentate nucleus and intravenous infusions [1][5] - The FALCON Phase 1b clinical trial is currently screening participants [1][3] FDA Designations - The Rare Pediatric Disease designation is for serious diseases affecting individuals under 18, potentially allowing the company to receive a pediatric priority review voucher (PRV) [2][4] - The PRV can expedite future Biologic License Applications and may be sold or transferred [2][4] Clinical Development - SGT-212 aims to restore therapeutic levels of the frataxin protein to address neurological, cardiac, and systemic manifestations of Friedreich's ataxia [1][5] - The therapy employs a dual route of administration, confirmed in real-time via MRI-enhancing contrast agent [5] Disease Overview - Friedreich's ataxia is a life-threatening degenerative disease caused by defects in the frataxin gene, affecting approximately 5,000 people in the U.S. and 15,000 in Europe [6] - The disease leads to progressive nervous system damage and cardiac dysfunction, with no current treatments available to halt its progression [6] Company Background - Solid Biosciences focuses on developing gene therapies for rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy and SGT-501 for catecholaminergic polymorphic ventricular tachycardia [7][8] - The company aims to improve the lives of patients with rare diseases through innovative genetic medicine [8]