AL050

AL050 is an investigational enzyme replacement therapy (ERT) being developed by Alector that targets glucocerebrosidase (GCase), a lysosomal enzyme. GCase is responsible for the hydrolysis of glucosylceramide into glucose and ceramide; mutations in the GBA1 gene, which encodes GCase, lead to a deficiency of this enzyme, resulting in Gaucher disease and serving as the most common genetic risk factor for Parkinson's disease. AL050 is designed to restore or enhance GCase activity to address lysosomal dysfunction and the toxic accumulation of substrates, which are implicated in the pathogenesis of various neurodegenerative conditions. The program is currently in the discovery or preclinical stage of development as part of Alector's immuno-neurology pipeline.

Other names

Alector Brain Carrier-GCaseABC-GCase