2019

GT Gain Therapeutics SA

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TARGET GENE

GBA1

TARGET ENZYME

Beta-glucocerebrosidase

PRIMARY

STORAGE

Alpha-synuclein

EPIDEMIOLOGY

9.1% of GBA carriers

UNMET MEDICAL NEED

Neurodegeneration, non motor symptoms, L-DOPA-induced dyskinesia and psychosis, motor fluctuation in L-DOPA therapy

DEVELOPMENT

STAGE

Discovery

In vitro/In vivo