VY-SOD101

VY-SOD101 is an investigational gene therapy developed by Voyager Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. The therapy utilizes a proprietary adeno-associated virus (AAV) capsid to deliver a microRNA (miRNA) expression cassette. This cassette is designed to leverage the RNA interference (RNAi) pathway to selectively silence the production of SOD1 messenger RNA, thereby reducing the levels of toxic mutant SOD1 protein in the central nervous system. Administered via a single intrathecal injection, VY-SOD101 aims to slow disease progression by targeting the underlying genetic cause of SOD1-mediated ALS. Preclinical studies have demonstrated significant knockdown of SOD1 expression in spinal cord motor neurons.