Solid Biosciences Inc., which acquired UF startup AavantiBio, announced that the first participant has been dosed in FALCON, the Company’s Phase 1b, first-in-human clinical trial evaluating SGT-212, its investigational gene therapy for the treatment of Friedreich’s ataxia (FA).
SGT-212 is a first-in-class investigational gene therapy for the treatment of FA that employs a dual-route administration designed to target the neurologic, cardiac and systemic manifestations of the disease, which are key drivers of morbidity and mortality seen in FA. SGT-212 is delivered through a precise, stereotactic, MRI-guided intradentate nuclei (IDN) infusion to the cerebellar dentate nuclei followed by an intravenous (IV) infusion, with the goal of restoring therapeutic levels of frataxin.
