Solid Biosciences Testing Dual Delivery Gene Therapy in First-in-Human Friedreich's Ataxia Trial

Precision Medicine Online. Apr 16, 2026. NEW YORK – Solid Biosciences is advancing its first-in-human trial of SGT-212, an experimental gene therapy for the rare neuromuscular disorder Friedreich's ataxia. 

The dual route of administration is intriguing to Kisanuki, since Friedreich's ataxia implicates multiple tissues in the body. "If we used just systemic delivery, we might not fully benefit the neuro phenotype," he said. "But if we just focus on brain delivery, we're ignoring the cardiac phenotype."

Solid Biosciences Testing Dual Delivery Gene Therapy in First-in-Human Friedreich's Ataxia Trial