Gabriel Brooks, MD, Chief Medical Officer at Solid Biosciences, and Russell Lonser, MD, of The Ohio State University Wexner Medical Center, discuss SGT-212 and the FALCON clinical trial for patients with Friedreich’s ataxia.
Friedreich’s ataxia is a progressive, genetic disorder that particularly affects the nervous system. Common features include the gradual loss of strength and sensation in the arms and legs, spasticity, and impaired speech. Hypertrophic cardiomyopathy, diabetes, impaired vision, hearing loss, and/or scoliosis may also develop in these individuals. Most people with Friedreich ataxia begin to experience the signs and symptoms in puberty. This condition is caused by genetic changes in the FXN gene that result in reduced levels of functional frataxin, a protein involved in several mitochondrial functions.
SGT-212 is an investigational recombinant AAV gene replacement therapy designed to deliver full-length human frataxin via a dual-route of administration designed to target the neurologic, cardiac, and systemic manifestations of Friedreich’s ataxia by restoring frataxin levels. The therapy is delivered through a precise, stereotactic, MRI-guided intradentate nuclei infusion to the cerebellar dentate nuclei followed by an intravenous infusion.
The FALCON clinical trial (NCT07180355) is a phase 1b first-in-human, open-label, multicenter study evaluating the safety and tolerability of SGT-212 in patients ages 18 to 40 years with Friedreich’s ataxia and cardiac hypertrophy. As Dr. Brooks explains, the first patient was dosed in early 2026 and next steps include continuation of dosing in early cohorts.
For more information, visit https://solidbio.com. Solid is a patient-focused organization founded and led by individuals directly impacted by Duchenne. Its mission is to improve the daily lives of patients living with devastating rare diseases.
To learn more about Friedreich’s ataxia and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/