Solid Biosciences and SGT-212 gene therapy updates

In people with FA, a protein called frataxin is reduced. SGT-212, developed by pharmaceutical company Solid Biosciences, is designed to deliver frataxin to both the cerebellum in the brain, and to the heart. SGT-212 is the only dual route gene therapy in development to treat FA.  

Update from January 2025
In January 2025 Solid Biosciences announced that they were granted an investigational new drug designation by the US drug regulators, the FDA, to trial SGT-212 in adults with FA. 

Update from 01/12/2025 
In December 2025, they announced that SGT-212, had been granted Rare Pediatric Disease and Fast Track designations by the FDA. Rare Pediatric Disease designation is granted by the FDA for serious and life-threatening conditions that primarily affect individuals under 18 years of age and is designed to incentivise and encourage the development of rare disease treatments for children. Fast track designation helps to accelerate drug development for serious conditions and unmet medical needs. Read the press release here . 

Update from 12/01/2026 
In January 2026 Solid Biosciences announced that they had dosed their first participant in their Phase 1b FALCON Trial which is being undertaken in the US. It is an open-label, multicentre Phase 1b trial evaluating the safety and tolerability of SGT-212 in people with FA and cardiac hypertrophy, aged 18 to 40 years. Read the press release here, and read more about the trial here. The FALCON trial is currently being carried out in the US only. 

On the same day, they also announced that they received FDA Orphan Drug designation for SGT-212 dual-route gene therapy for the treatment of FA. Receiving Orphan Drug Designation (ODD) means that a number of incentives are granted with the aim of encouraging and accelerating the drug’s development process. Read the press release here.