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AVB-202-TT

Development stage: Preclinical
Lead developer: Solid Biosciences
Modality: Gene Therapies
Administration: Intravenous, Intrathecal

Overview

AVB-202-TT is a preclinical gene therapy candidate developed for the treatment of Friedreich’s ataxia, a rare hereditary neurodegenerative disorder. Originally developed by AavantiBio (which was acquired by Solid Biosciences in 2022), the program utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the frataxin (FXN) gene to affected tissues, including the heart and nervous system. The therapy aims to restore frataxin protein levels, thereby addressing the underlying mitochondrial dysfunction and oxidative stress characteristic of the disease. The program originated from research at the University of Florida.

Other names: AVB-202AVB202AVB 202

Targets

FXN (Frataxin)

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