Shots:
- The US FDA has granted FTD to SGT-212 for treating Friedreich’s ataxia (FA) and previously approved its IND on Jan 07, 2025
- The P-Ib study will assess safety & tolerability of contemporaneous systemic IV and bilateral IDN administration of SGT-212 in non-ambulatory and ambulatory FA patients for 5yrs., with dosing anticipated during H2’25
- SGT-212 is a recombinant AAV-based gene therapy that transfers full-length human frataxin via intradentate nucleus (IDN) in the cerebellar dentate nuclei & IV infusion in the cardiomyocytes. The IDN delivery will be confirmed in real-time using gadolinium MRI contrast agent
Ref: Solid Biosciences | Image: Solid Biosciences
Related News:- Solid Biosciences Signed an Exclusive License Agreement with Armatus Bio for AAV-SLB101 in Facioscapulohumeral Muscular Dystrophy (FSHD)
PharmaShots! Your go-to media platform for customized news ranging for multiple indications. For more information connect with us at connect@pharmashots.com
