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AGIL-FA
- Development stage
- Discontinued
- Lead developer
- PTC Therapeutics
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
AGIL-FA is a gene therapy developed for the treatment of Friedreich's ataxia (FA). It consists of a gene construct delivered into the central nervous system using adeno-associated virus serotype 5 (AAV5) technology. The therapy aims to restore lost function in the frataxin (FXN) gene by delivering corrective DNA to specific CNS cells, enabling safe and effective long-term expression of the frataxin protein. Friedreich's ataxia is a neurodegenerative disease caused by a defect in the FXN gene that reduces production of the frataxin protein.
- Other names
- Recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein
02
Targets
FXN (Frataxin)
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