Posted by Michael Wonder on 02 Dec 2025
Solid Biosciences receives FDA rare paediatric disease designation for SGT-212 dual route of administration gene therapy for Friedreich’s ataxia
1 December 2025 - Solid Biosciences today announced that it received rare paediatric disease designation from the US FDA 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, utilising 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 Friedreich’s ataxia.
