Solid Biosciences (SLDB) received Rare Pediatric Disease designation from the U.S. Food and Drug Administration for SGT-212, the Company’s investigational gene therapy for Friedreich’s ataxia. SGT-212 will deliver the full-length frataxin gene via dual routes of administration, utilizing both direct intradentate nucleus and intravenous infusions, and was designed to promote restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA.
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