2019

GT Gain Therapeutics SA

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TARGET GENE

GBA1

TARGET ENZYME

Beta-glucocerebrosidase

PRIMARY

STORAGE

Glucosylceramide

SECONDARY

STORAGE

Sphingosine &

alpha-synuclein

EPIDEMIOLOGY

1 : 40-60,000

UNMET MEDICAL NEED

Neurological and bone diseases, pulmonary manifestations, growth rate, cancers, few oral drugs

DEVELOPMENT

STAGE

Discovery

In vitro