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Solid Biosciences Announces FDA IND Clearance for First-In-Industry Dual Route of Administration Gene Therapy to Treat Both Neurologic and Cardiac Manifestations of Friedreich’s Ataxia
- SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the CNS and cardiac manifestations of Friedreich’s ataxia - - Dual
About this update from Solid Biosciences Inc.
[{"type":"text","content":"- SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the CNS and cardiac manifestations of Friedreich’s ataxia - - Dual route of administration enables direct delivery of AAV-based gene therapy to the cerebellum and heart to potentially address the most significant symptoms of the disease - - Phase 1b clinical trial initiation expected in 2H 2025 - - Company to hold a conference call tomorrow, January 8, 2025, at 8:30 AM ET - CHARLESTOWN, Mass., Jan. 07, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for SGT-212 for the treatment of Friedreich’s ataxia (FA), a degenerative disease caused by insufficient levels of the frataxin protein. SGT-212 is the Company’s novel, AAV-based FA gene therapy candidate designed to deliver full-length frataxin via systemic intravenous (IV) infusion as well as direct intradentate nuclei (IDN) infusion into the cerebellum. SGT-212 is designed to treat the neurologic and systemic clinical manifestations of FA to address the full spectrum of disease progression. FA is a highly complex, multisystem disease that presents distinct challenges for drug development, in part because frataxin is a protein that requires: (1) precise expression levels to avoid fatal cardiac toxicities, and (2) on-target tissue localization in the cerebellum to achieve potential neurological clinical benefit. SGT-212 is the only candidate in development using two routes of administration to address the cardiac manifestations of FA while also directly delivering therapy to the dentate nuclei in the cerebellum, the region most affected and implicated in FA-associated neurologic decline. Bo Cumbo, President and CEO of Solid Biosciences, commented: “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 m...