ATXN2 microRNA gene therapy

ATXN2 microRNA gene therapy is an investigational therapeutic approach designed to treat amyotrophic lateral sclerosis (ALS) by reducing the expression of the ATXN2 gene. Developed by Maze Therapeutics using its proprietary COMPASS platform, the therapy employs an adeno-associated virus (AAV) vector to deliver microRNA (miRNA) sequences that specifically target and degrade ATXN2 messenger RNA. This reduction in ataxin-2 protein levels is intended to mitigate the toxic aggregation of TDP-43, a protein found in pathological clusters in up to 97% of ALS patients. By lowering ATXN2 levels, which acts as a potent genetic modifier of TDP-43 toxicity, the therapy aims to slow or halt disease progression in both sporadic and familial forms of ALS. As of early 2022, the program was in the preclinical stages of development.

Other names

ATXN2-targeting AAV-microRNAATXN-2-targeting AAV-microRNAATXN 2-targeting AAV-microRNAataxin-2 gene therapyataxin2 gene therapyataxin 2 gene therapyAAV-miRNA-ATXN2AAV-miRNA-ATXN-2AAV-miRNA-ATXN 2