Early signals stack up: Two small molecules activate GCase in Parkinson’s

Gain Therapeutics’ new Phase Ib data mark the second report in as many months showing that GCase— a lysosomal enzyme genetically and mechanistically tied to Parkinson’s disease — can be activated by small molecules in patients. The update follows similar early findings from Vanqua Bio, using a different biomarker approach.

The symptoms of Parkinson’s disease and their progression are driven by loss of the dopamine-producing cells in the brain. What drives that loss has been the central question. Lysosomal dysfunction has emerged as a leading hypothesis, propelled by a clustering of genetic risk factors, such as the drug target LRRK2, in endosomal and lysosomal pathways. ...