Delivering Gene Therapy’s Promise
To improve the lives of children with CNS diseases
Click to learn more
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 Enters into an Exclusive Worldwide License Agreement with SATT Conectus for a Gene Therapy Candidate for the Treatment of the Fragile X Syndrome

 Lysogene Enters into an Exclusive Worldwide License Agreement with SATT Conectus for a Gene Therapy Candidate for the Treatment of the Fragile X Syndrome 150 150 Lysogene

Strengthening of Lysogene’s pipeline with a new drug candidate for a pathology with high unmet…

Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis

 Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis 150 150 Lysogene

Second Lysogene CNS gene therapy program to enter the clinic Ongoing enrollment of a total…

LYSOGENE – Meeting Notice of the Annual Ordinary and Extraordinary General Meeting to be held on 25 June 2021

 LYSOGENE – Meeting Notice of the Annual Ordinary and Extraordinary General Meeting to be held on 25 June 2021 150 150 Lysogene

Paris, France — 20 May 2021 at 06:00 pm CEST — Lysogene (FR0013233475 – LYS),…

Lysogene Reports its Cash Position as of 31 March 2021

 Lysogene Reports its Cash Position as of 31 March 2021 150 150 Lysogene

Cash and cash equivalents of €20.0 million* as of 31 March 2021 Paris, France —…

Lysogene Announces Filing of 2020 Universal Registration Document

 Lysogene Announces Filing of 2020 Universal Registration Document 150 150 Lysogene

Paris, France — 13 April 2021 at 06:00pm — Lysogene (FR0013233475 – LYS), a phase…

Lysogene Reports Full Year 2020 Financial Results and Provides Operational Update

 Lysogene Reports Full Year 2020 Financial Results and Provides Operational Update 150 150 Lysogene

Positive biomarker data reported with LYS-SAF302 for the treatment of MPS IIIA New program entering…

Lysogene Reports its Cash Position as of 31 December 2020

 Lysogene Reports its Cash Position as of 31 December 2020 150 150 Lysogene

Cash and cash equivalents of €18.8 million* as of 31 December 2020 Paris, France —…

Lysogene Reports LYS-SAF302 Biomarker Data Presented at the WORLDSymposium™ 2021

 Lysogene Reports LYS-SAF302 Biomarker Data Presented at the WORLDSymposium™ 2021 150 150 Lysogene

Evidence of positive biological response to LYS-SAF302 in patients treated in the AAVance clinical trial…

Lysogene Receives FDA Clearance of Investigational New Drug Application to Initiate the Gene Therapy Clinical Trial in the US with LYS-GM101 for the Treatment of GM1 Gangliosidosis

 Lysogene Receives FDA Clearance of Investigational New Drug Application to Initiate the Gene Therapy Clinical Trial in the US with LYS-GM101 for the Treatment of GM1 Gangliosidosis 150 150 Lysogene

Second country to authorize trial initiation after recent UK MHRA approval, further country approvals pending…

Lysogene Secures €5 million in Non-Dilutive Financing

 Lysogene Secures €5 million in Non-Dilutive Financing 150 150 Lysogene

Approval received from BRED for a €5 million non-dilutive financing in the form of PGE…

All News

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.

Learn more
GM1 Gangliosidosis

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

Learn more
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.

Learn more
Gaucher & Parkinson's

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

Learn more

Lysogene COVID-19 update

Find out More

Lysogene is developing therapies for children with CNS diseases.

Delivering Gene Therapy’s Promise

About Us
Our Science
Meet the Team
Diseases
Resources

Contact Us
Careers
Transparency
Compassionate Use Policy

©2020 Lysogene. All rights reserved.

Cookie Policy
Privacy Policy
Legal Notice

Get In Touch

Don’t hesitate to contact us if you need more help.

    Back to top