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SGT-212
- Development stage
- Phase 1
- Lead developer
- Solid Biosciences
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Recombinant Proteins and Enzymes
- Administration
- Intracerebral, Intravenous
01
Overview
SGT-212 is a recombinant adeno-associated virus (AAV)-based gene replacement therapy in development for Friedreich's ataxia (FA). It is designed to deliver full-length human frataxin (FXN) via a dual route of administration—direct intradentate nucleus (IDN) infusion into the cerebellum using an MRI-guided device, and systemic intravenous (IV) infusion. This approach aims to increase therapeutic FXN levels in both the cerebellar dentate nuclei and cardiomyocytes, addressing the neurological and cardiac manifestations of FA. Restoration of FXN levels is expected to repair mitochondrial dysfunction in neurons and cardiomyocytes, potentially treating both neurologic decline and cardiac complications associated with FA. SGT-212 is currently being developed by Solid Biosciences, with clinical trials planned to begin in 2025[1][2][3][4][5][6][7][8][9].
- Other names
- FA212FA-212FA 212
02
Targets
FXN (Frataxin)
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