A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

NCTID	NCT07180355 (View at clinicaltrials.gov)
Description	This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA. All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years. (Show More)
Development Status 🔄	Active
Indication 🔄	Friedreich's Ataxia
Disease Ontology Term 🔄	DOID:0111218
Compound Name 🔄	SGT-212
Sponsor	Solid Biosciences Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	10 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant 🔄	FXN
Therapy Type 🔄	Gene transfer
Therapy Route 🔄	In-vivo
Mechanism of Action 🔄	Functional gene replacement
Route of Administration 🔄	Intradentate nucleus, intravenous
Drug Product Type 🔄	Viral vector
Delivery System 🔄	Viral transduction
Vector Type 🔄	AAVhu68
Dose 1 🔄	Undisclosed

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations 🔄	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	First patient dosed January 2026, initial data expected H2 2026