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
About Lysogene
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 technology
Our robust pipeline

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

Pipeline

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.

Clinical Trials

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.

Clinical Trials

Latest News

News, press releases, and more.

Lysogene Announces Favorable DSMB Review Recommending Study Continuation and Positive Biomarker Data in Stage 1 of Adaptative Clinical Trial with LYS-GM101 Gene Therapy in GM1 Gangliosidosis

 Lysogene Announces Favorable DSMB Review Recommending Study Continuation and Positive Biomarker Data in Stage 1 of Adaptative Clinical Trial with LYS-GM101 Gene Therapy in GM1 Gangliosidosis 150 150 Lysogene

Completion of treatment and 6 months follow-up for the last patient enrolled in Stage 1…

Lysogene Provides Updates and Topline Results from Phase 2/3 AAVance Gene Therapy Clinical Study

 Lysogene Provides Updates and Topline Results from Phase 2/3 AAVance Gene Therapy Clinical Study 150 150 Lysogene

In the cohort of patients enrolled before the age of 30 months, a statistically significant…

Lysogene to Provide Updates and Topline Results from Phase 2/3 AAVance Gene Therapy Clinical Study and Host Webcast on Wednesday, November 23, 2022

 Lysogene to Provide Updates and Topline Results from Phase 2/3 AAVance Gene Therapy Clinical Study and Host Webcast on Wednesday, November 23, 2022 150 150 Lysogene

Live webcast to be hosted on Wednesday, November 23, 2022 at 1:30 pm CET Paris,…

Lysogene Reports its Cash Position as of 30 September 2022

 Lysogene Reports its Cash Position as of 30 September 2022 150 150 Lysogene

Cash and cash equivalents of €6.1 million[1] as of 30 September 2022 Paris, France —…

Lysogene Reports First Half 2022 Financial Results

 Lysogene Reports First Half 2022 Financial Results 150 150 Lysogene

Full data analysis of AAVance Phase 2/3 Gene Therapy Clinical Trial with LYS-SAF302 in MPS…

Lysogene: New Board of Directors

 Lysogene: New Board of Directors 150 150 Lysogene

Paris, France —16 September 2022 at 8:00 am CET — Lysogene (FR0013233475 – LYS), a…

Lysogene Reports its Cash Position as of 30 June 2022

 Lysogene Reports its Cash Position as of 30 June 2022 150 150 Lysogene

Cash and cash equivalents of €7.7 million[1] as of 30 June 2022 Paris, France —…

Half-year liquidity contract statement for LYSOGENE

 Half-year liquidity contract statement for LYSOGENE 150 150 Lysogene

  Paris, France —11 July 2022 at 06:00 pm — Lysogene (FR0013233475 – LYS) —…

Lysogene Provides Additional Update on AAVance Phase 2/3 Gene Therapy Clinical Trial with LYS-SAF302 in children with MPS IIIA

 Lysogene Provides Additional Update on AAVance Phase 2/3 Gene Therapy Clinical Trial with LYS-SAF302 in children with MPS IIIA 150 150 Lysogene

• Positive biomarker data confirmed in additional subjects and at additional timepoints • Stable or…

Lysogene releases results from June 24,2022, ordinary and extraordinary annual general meeting. Shareholders approved all proposed resolutions.

 Lysogene releases results from June 24,2022, ordinary and extraordinary annual general meeting. Shareholders approved all proposed resolutions. 150 150 Lysogene

  Paris, France —27 June 2022 at 8:00 am CET — Lysogene (FR0013233475 – LYS),…

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Diseases

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

MPS IIIA
GM1 Gangliosidosis
Fragile X
Gaucher & Parkinson's
MPS IIIA

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.

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GM1 Gangliosidosis

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

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Fragile X

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.

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Gaucher & Parkinson's

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

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Company Award for Innovation

The EURORDIS Company Award for Innovation recognizes Lysogene as a pioneering gene therapy company which has established itself among major players driving European-led innovation, in just over ten years.

#EURORDISAWARDS2022

Lysogene COVID-19 update

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Lysogene is developing therapies for children with CNS diseases.

Delivering Gene Therapy’s Promise

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