2019

GT Gain Therapeutics SA

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TARGET GENE

GLB1

TARGET ENZYME

Acid beta-galactosidase

PRIMARY

STORAGE

GM1 ganglioside & keratan sulfate

SECONDARY

STORAGE

Alpha-synuclein

EPIDEMIOLOGY

1 : 100-200,000

UNMET MEDICAL NEED

No available treatment or cure

DEVELOPMENT

STAGE

Preclinical

Lead Optimization