Delivering Gene Therapy’s Promise
To improve the lives of children with CNS diseases
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Lysogene’s determination to succeed is rooted in our deep understanding of the impact central nervous system (CNS) diseases have on patients and families, and springs from our founder whose own child was affected by a neurodegenerative disease.
Our best-in-class technology

Pioneering gene therapies for CNS disorders

Our advanced gene therapy technology capitalizes on our differentiated knowledge of direct-to-brain administration of AAV vectors, allowing us to precisely target the causes of disease.

Our robust pipeline

Extraordinary progress in developing therapies for CNS diseases with lead programs in neurodegenerative lysosomal storage disorders.

Transforming the Lives
of Patients and Families

Our team is committed to developing pioneering therapies that will transform the lives of children affected by these devastating diseases.

Transforming the Lives
of Patients and Families

Our team is committed to fulfilling gene therapy’s potential by developing pioneering therapies that will transform the lives of children affected by these devastating diseases.

Latest News

News, press releases, and more.

Diseases

Valuable information designed to provide knowledge and insights to patients, families, and caregivers.

Mucopolysaccharidosis type IIIA (MPS IIIA), or Sanfilippo syndrome type A, is a rare neurodegenerative lysosomal storage disorder characterized by intractable behavioral problems and developmental regression that typically appears in infancy or early childhood.

GM1 gangliosidosis, or Landing disease, is a rare inherited neurodegenerative lysosomal storage disorder characterized by severe cognitive and motor developmental delays.

Fragile X syndrome (FXS) is the most common inherited cause of autism spectrum disorder and affects approximately 1 in 4,000 to 5,000 boys and 1 in 8,000 girls.

Neuronopathic Gaucher and Parkinson’s disease are both associated with mutations in the GBA1 gene and cause neurological symptoms.

Lysogene COVID-19 update

Get In Touch

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