Solid Biosciences's IND For Gene Therapy To Treat Friedreich's Ataxia Gets FDA Clearance

(RTTNews) - Life sciences company Solid Biosciences Inc. (SLDB), Tuesday 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.

In the second half of 2025, the company expects to initiate a first-in-human, open-label, dose-finding Phase 1b clinical trial of SGT-212. The study will enroll non-ambulatory and ambulatory adult patients living with FA across up to three cohorts and will evaluate the safety and tolerability of contemporaneous systemic and bilateral IDN administration of SGT-212. Participants in the trial will be followed out to five years after receiving SGT-212.