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 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),…

Lysogene Annual Ordinary and Extraordinary General Meeting of 24 June 2022 Availability of the Preparatory Documents

 Lysogene Annual Ordinary and Extraordinary General Meeting of 24 June 2022 Availability of the Preparatory Documents 150 150 Lysogene

  Paris, France —03 June 2022 at 6:00 pm CET — Lysogene (FR0013233475 – LYS),…

Lysogene Provides Clinical Update on AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Treatment of MPS IIIA at the 25th Annual ASGCT Meeting

 Lysogene Provides Clinical Update on AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Treatment of MPS IIIA at the 25th Annual ASGCT Meeting 150 150 Lysogene

• Promising efficacy data in young patients with persistent increase or stabilization in 3 main…

Lysogene Reports its Cash Position as of 31 March 2022

 Lysogene Reports its Cash Position as of 31 March 2022 150 150 Lysogene

Cash and cash equivalents of €10.7 million[1] as of 31 March 2022 Paris, France —…

Lysogene Publishes Positive Preliminary Preclinical Results in the Treatment of Fragile X Syndrome

 Lysogene Publishes Positive Preliminary Preclinical Results in the Treatment of Fragile X Syndrome 150 150 Lysogene

Paris, France — 21 April 2022 at 08:00 am CEST — Lysogene (FR0013233475 – LYS),…

Lysogene Announces Filing of 2021 Universal Registration Document

 Lysogene Announces Filing of 2021 Universal Registration Document 150 150 Lysogene

Paris, France — 20 April 2022 at 08:00 am — Lysogene (FR0013233475 – LYS), a…

Lysogene Enters into an Exclusive Worldwide License Agreement with Yeda, the commercial arm of the Weizmann Institute of Science, for a Novel Gene Therapy Candidate for Neuronopathic Gaucher Disease and Parkinson Disease with GBA1 Mutations

 Lysogene Enters into an Exclusive Worldwide License Agreement with Yeda, the commercial arm of the Weizmann Institute of Science, for a Novel Gene Therapy Candidate for Neuronopathic Gaucher Disease and Parkinson Disease with GBA1 Mutations 150 150 Lysogene

Lysogene exercises its option after a conclusive research collaboration period Paris, France — 12 April 2022…

Lysogene Reports Full Year 2021 Financial Results

 Lysogene Reports Full Year 2021 Financial Results 150 150 Lysogene

Paris, France – 31 March 2022 at 8:00 am CET – Lysogene (FR0013233475 – LYS),…

Lysogene Provides Update on Efficacy, Safety and Timelines of the AAVance phase 2/3 clinical trial with gene therapy LYS-SAF302 for the treatment of MPSIIIA

 Lysogene Provides Update on Efficacy, Safety and Timelines of the AAVance phase 2/3 clinical trial with gene therapy LYS-SAF302 for the treatment of MPSIIIA 150 150 Lysogene

Topline data of the main cohort of AAVance clinical trial expected in Q3 2022 Stabilization…

Lysogene Reports Additional Positive Biomarker data with LYS-SAF302 and Favorable Safety data with LYS-GM101 presented at the WORLDSymposium™ 2022

 Lysogene Reports Additional Positive Biomarker data with LYS-SAF302 and Favorable Safety data with LYS-GM101 presented at the WORLDSymposium™ 2022 150 150 Lysogene

New positive biomarker data from 16 patients treated with LYS-SAF302 showing reduction in heparan sulfate…

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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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