Solid Biosciences Says European Commission Grants Orphan Designation To SGT-212 For Treatment Of Friedreich’s Ataxia

- Obtaining Orphan designation from the European Commission reflects Solid’s commitment to the global development of SGT-212 for individuals living with Friedreich’s ataxia -

- SGT-212 is a first-in-class dual route FA gene therapy designed to combine direct, MRI-guided delivery to the dentate nuclei with systemic delivery to reach cardiac and other systemic tissues -

- Five participants have been dosed in the ongoing Phase 1b FALCON clinical trial and SGT-212 has been well tolerated as of October 5, 2026 -

- Solid expects to report initial FALCON clinical data in Q1 2027 -

CHARLESTOWN, Mass., Oct. 26, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (NASDAQ:SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that the European Commission (EC), acting upon the positive opinion of the European Medicines Agency’s Committee for Orphan Medicinal Products, has granted Orphan designation to SGT-212 for the treatment of Friedreich’s ataxia (FA). Solid also announced that five participants have received SGT-212 in the Phase 1b FALCON clinical trial, with no treatment-related serious adverse events observed as of October 5, 2026.

SGT-212 is a novel investigational gene therapy specifically designed to address the multisystem biological impact of FA. Its dual-route administration combines precise, MRI-guided delivery directly to the cerebellar dentate nuclei with systemic intravenous delivery intended to reach the heart and other affected systemic tissues. Degeneration of the dentate nuclei, key sites of neurologic pathology in FA, disrupts coordinated movement and contributes to progressive loss of balance, limb control and oculomotor function as well as to bulbar dysfunction, including declines in speech and swallowing. Through this dual-route approach, SGT-212 is designed with the expectation of delivering the full-length frataxin gene to the tissues known to drive disease-specific morbidity and mortality, with the intent to restore mitochondrial health for patients living with FA.