Solid Biosciences (NASDAQ:SLBD) announced that the FDA has cleared its IND application for SGT-212, a first-in-industry dual-route of administration gene therapy designed to treat the neurologic and cardiac manifestations of Friedreich’s ataxia (FD).
The company explained that FA is a highly complex, multisystem disease that poses unique challenges for drug development. Addressing these challenges requires frataxin, a protein, to achieve: (1) precise expression levels to avoid life-threatening cardiac toxicities, and (2) targeted tissue localization in the cerebellum to deliver neurological benefits.
Solid plans to initiate a first-in-human, open-label, dose-finding Phase 1b clinical trial of SGT-212 in the second half of 2025.
In a statement, Bo Cumbo, president and CEO of Solid, said, “SGT-212 has been intentionally designed to enable highly targeted delivery of our gene therapy to both the dentate nuclei and cardiac tissue. The IND was supported by a robust preclinical package demonstrating safe transduction and frataxin expression in these target tissues, with significant restoration of neurologic function and reversal of the cardiac implications of the disorder in mice.”
“Over the years, we have tested several candidates using different methods of administration and have conducted multiple NHP studies, some of which extended out to a year. Based on this research, we believe a dual route of administration targeting multiple systems is the best approach in development to directly address the neurological implications that profoundly impact the everyday life of patients, while simultaneously targeting the cardiac manifestations that play a key role in more progressed disease. SGT-212 offers a truly differentiated approach to addressing FA with the potential to treat the full spectrum of symptoms, and we hope to meet each patient where they are in their FA disease course,” Mr. Cumbo added.
