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

CHARLESTOWN, Mass., Dec. 01, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. today announced that it received Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) for SGT-212, the Company’s investigational gene therapy for Friedreich’s ataxia (FA). SGT-212 will deliver the full-length frataxin gene via dual routes of administration, utilizing both direct intradentate nucleus (IDN) and intravenous (IV) infusions, and was designed to promote restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA.

Together with the Fast Track designation granted earlier this year, it recognizes our dual-route clinical approach for FALCON, our first-in-human trial, which is now screening participants, as an important first step in meeting an unmet need for FA. These designations are designed to help accelerate time to market and enhance engagement with the FDA. We look forward to continued collaboration with regulators to bring this therapy to patients as quickly as possible.

Enhancing the Objective Assessment of Friedreich Ataxia Severity: A Multiview IMU-Based Approach

Ranaweera K, Nguyen BA, Pathirana PN, Milne SC, Horne M, Delatycki MB, Corben LA. Enhancing the Objective Assessment of Friedreich Ataxia Severity: A Multiview IMU-Based Approach. Annu Int Conf IEEE Eng Med Biol Soc. 2025 Jul;2025:1-6. doi: 10.1109/EMBC58623.2025.11253596. PMID: 41335798. 

These findings indicate that multiview IMU-based systems can provide sensitive and reliable assessments of severity of ataxia in FRDA.Clinical relevance-This study presents a multiview IMU-based approach that can enhances the objective assessment of severity of ataxia in FRDA.