12:00 AM
May 26, 2014
 |  BioCentury  |  Emerging Company Profile

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 the enzyme is deficient or defective.

In 2011, co-founder Dimitri Krainc published work in Cellshowing the functional loss of GCase impairs lysosomal protein degradation and can lead to the accumulation of alpha-synuclein (SNCA), a protein linked to Parkinson's.

The exact link between Gaucher's and Parkinson's is not well understood, but as lysosomal GCase activity goes down in Gaucher's patients, SNCA levels go up.

Krainc is a professor at Northwestern University Feinberg School of Medicine and director of the school's Center for Rare...

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