India, Oct. 26 -- Solid Biosciences Inc. (SLDB) announced that the European Commission has granted Orphan Medicinal Product designation to SGT-212, an investigational gene therapy being developed to treat Friedreich's ataxia (FA), a rare inherited disease that causes progressive neurological damage and cardiac dysfunction.
The company also reported continued progress in the Phase 1b FALCON clinical trial, with five participants dosed as of October 5, 2026.
No treatment-related serious adverse events had been observed at that time.
SGT-212 is designed to deliver the full-length frataxin gene through a dual-route approach, combining MRI-guided delivery to the cerebellar dentate nuclei with intravenous administration intended to reach the h...