CO-1 (Congruence Therapeutics)

CO-1 is an experimental small molecule activator being developed by Congruence Therapeutics for the treatment of Parkinson's Disease associated with mutations in the GBA1 gene (GBA-PD). It is designed to enhance the activity of the lysosomal enzyme glucocerebrosidase (GCase). In patients with GBA-PD, mutations in the GBA1 gene lead to deficient GCase activity, which causes lysosomal dysfunction and the pathological accumulation of alpha-synuclein. CO-1 was discovered using Congruence's ReSSeS (Resolving Structure in Solution) computational platform, which identifies novel binding sites by modeling protein conformational dynamics. By restoring GCase activity, CO-1 aims to improve lysosomal function and potentially modify the course of the disease. The program is currently in the preclinical stage of development.