Lysosomal: Activating GCase

Lysosomal developing allosteric GCase modulators for PD, synucleinopathies

Lysosomal Therapeutics Inc. is developing brain-penetrant small molecules for a genetically validated Parkinson's target that it believes will be disease-modifying for idiopathic PD and potentially other synucleinopathies.

The biotech has a license from NIH to allosteric modulators that increase glucocerebrosidase (GBA; GCase) activity in the lysosome. Lysosomal expects its recent $4.8 million seed round will provide the runway to generate lead-stage compounds in the next 12-18 months.

GCase is best known for its role in Gaucher's disease - a genetic lysosomal storage disorder in which

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