AXO-AAV-FTD

**AXO-AAV-FTD** is an investigational AAV gene therapy developed using Silence-and-Replace technology for frontotemporal dementia (FTD) associated with C9orf72 gene mutations. It employs a single AAV vector to deliver DNA-directed RNA interference (ddRNAi) to silence mutant C9orf72 alleles while simultaneously expressing a functional copy of the wild-type C9orf72 gene, aiming to reduce toxic gain-of-function effects and restore normal protein levels in the central nervous system. Developed through a collaboration between Axovant Gene Therapies and Benitec Biopharma as part of a broader platform for autosomal dominant disorders, it targets the underlying genetic cause of FTD and amyotrophic lateral sclerosis (ALS), with preclinical data supporting its potential in nucleotide repeat expansion diseases.