pariceract

Pariceract is a first-in-class small-molecule activator of the lysosomal enzyme glucocerebrosidase (GCase), developed for the treatment of Parkinson’s disease associated with pathogenic variants in the glucocerebrosidase 1 (GBA1) gene (GBA-PD). It readily crosses the blood–brain barrier and engages GCase within the central nervous system to enhance residual enzyme activity and improve lysosomal function, aiming to reduce alpha-synuclein accumulation and modify disease progression in GBA-PD. The drug originated from Lysosomal Therapeutics (as LTI-291) and is being clinically developed by Bial, which has advanced it through early clinical trials in healthy volunteers and GBA-PD patients and is preparing more advanced phase 2 studies to assess its ability to delay clinically meaningful motor progression in this genetically defined Parkinson’s disease population.

Other names

Pariceract