Solid Biosciences Receives FDA Clearance for Gene Therapy to Treat Friedreich’s Ataxia
Wednesday, January 08, 2025
Solid Biosciences has announced that its Investigational New Drug (IND) application for SGT-212 has been cleared by the U.S. Food and Drug Administration (FDA).
FA affects approximately 5,000 individuals in the United States and 15,000 in Europe. It is an inherited, life-threatening disorder caused by defects in the frataxin gene, leading to progressive nervous system and cardiac dysfunction.
Currently, there are no treatments that cure or halt the progression of FA, highlighting the significant unmet need for innovative therapies like SGT-212.
This gene therapy candidate is designed to address Friedreich’s ataxia (FA), a degenerative disease resulting from insufficient levels of the frataxin protein.
SGT-212 was developed through a collaboration involving FA212 LLC, the University of Pennsylvania, and Solid Biosciences. The therapy uses a recombinant AAV-based gene replacement approach to deliver full-length human frataxin.
This dual-administration method seeks to restore mitochondrial function in neurons and cardiomyocytes, addressing both neurological and cardiac symptoms of the disease.
SGT-212 employs a dual route of administration, delivering therapy both intravenously and directly into the cerebellar dentate nuclei via intradentate nuclei (IDN) infusion.
This approach aims to address both the neurological and cardiac symptoms associated with the disease, offering a more comprehensive treatment strategy.
FA is a complex, multisystem condition that presents significant challenges for treatment development. It requires precise delivery of the frataxin protein to avoid cardiac toxicity while also targeting the cerebellum to provide neurological benefits. SGT-212 is the first therapy in development designed to address these challenges through its dual-administration approach, focusing on the dentate nuclei for neurological treatment and cardiac tissue for systemic manifestations.
Preclinical studies have shown promising results, demonstrating safe delivery of frataxin to target tissues, improved neurological function, and reversal of cardiac symptoms in animal models. These findings support the dual-route methodology as a potentially effective treatment for FA.
A Phase 1b clinical trial for SGT-212 is expected to commence in the second half of 2025. The trial will involve ambulatory and non-ambulatory adult patients with FA, assessing safety and tolerability across up to three patient cohorts. Participants will be monitored for five years following treatment.
This milestone reflects years of collaborative research and development involving patient advocacy groups, research institutions, and academic partners, aiming to improve outcomes for individuals living with FA.
Source: solidbio.com
