NMRA-GCase

**NMRA-GCase** is a preclinical small molecule program developed by Neumora Therapeutics that activates both wild-type and mutant forms of the glucocerebrosidase (GCase) enzyme, encoded by the GBA1 gene. Mutations in GBA1 occur in approximately 10% of Parkinson's disease (PD) patients and lead to lysosomal dysfunction, alpha-synuclein accumulation, neurotoxicity, and inflammation. By directly binding and enhancing GCase activity, NMRA-GCase aims to restore lysosomal function and mitigate PD pathology, addressing a key genetic risk factor with high-throughput screened activators showing potent biophysical engagement. The program has exclusively licensed intellectual property from Amgen and is advancing toward clinical development for PD.[1][3][5][10]

Other names

NMRA-GBA001NMRA-GBA-001NMRA-GBA 001