Efficacy and Safety of a Novel FXN Gene Therapy (AVB-202) for the Treatment of Friedreich’s Ataxia

169 Efficacy and Safety of a Novel FXN Gene Therapy (AVB-202) for the Treatment of Friedreich’s Ataxia; Grace K. Pavlath, Jennifer Wheeler, Arden Bond, Jennifer L. Marlowe.(Solid Biosciences); May 01, 2023 Volume 31Issue 4Supplement 1S1-794 26th Annual Meeting of the American Society of Gene & Cell Therapy 

 AVB-202, a novel AAV9 gene therapy product expressing FXN under the control of a CBA promoter, is intended to restore functional levels of frataxin across disease-relevant tissues using a dual route of administration (intravenous and intrathecal) with the goal of preventing progression or reversing cardiac and CNS manifestations of FA.

Efficacy and Safety of a Novel FXN Gene Therapy (AVB-202) for the Treatment of Friedreich’s Ataxia