At Lysogene, we’re on an urgent mission to find cures for diseases of the central nervous system with a clear focus on neurodegenerative lysosomal storage disorders. These rare, debilitating, and progressive diseases primarily affect children—their entire bodies and especially their brains.

Together with our global community of physicians, academics, patients, and families, we are committed to developing groundbreaking treatments that will transform the lives of people living with these diseases.

Our team is currently focused on research, development, and clinical trials for the following diseases:


GM1 Gangliosidosis

Fragile X Syndrome

Neuronopathic Gaucher and Parkinson's

Lysogene COVID-19 update

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