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

Solid Biosciences Inc., a biotechnology company that acquired UF startup AavantiBio, 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.

Read more about 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.