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

GlobeNewswire Inc. • Dec 1, 2025 8:00am (New York) •By Solid Biosciences Inc.

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

Solid Biosciences received FDA Rare Pediatric Disease designation for SGT-212, a dual-route gene therapy targeting Friedreich's ataxia. The therapy aims to restore frataxin protein levels and address neurologic, cardiac, and systemic disease manifestations.

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Company received important FDA designations (Rare Pediatric Disease and Fast Track) for its investigational gene therapy, indicating promising progress in developing a treatment for a rare genetic disorder

Keywords

#gene therapy #Friedreich's ataxia #FDA designation #rare disease

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