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 termination of license agreement with Sarepta for LYS-SAF302 program, regaining global commercial rights

 Lysogene announces termination of license agreement with Sarepta for LYS-SAF302 program, regaining global commercial rights 150 150 Lysogene

Lysogene remains focused on developing its pipeline led by LYS-SAF302, LYS-GM101, Fragile X and other…

Half-Year report on the liquidity contract signed by Lysogene with Kepler Cheuvreux

 Half-Year report on the liquidity contract signed by Lysogene with Kepler Cheuvreux 150 150 Lysogene

Paris, France — 5 January 2022 at 06:00pm CEST — Lysogene (FR0013233475 – LYS), a…

Lysogene Enters into a Loan Agreement of €15 million with the European Investment Bank (EIB)

 Lysogene Enters into a Loan Agreement of €15 million with the European Investment Bank (EIB) 150 150 Lysogene

The financing will enable Lysogene to accelerate the development of its gene therapy platform The…

Lysogene awarded Innovation Passport by the UK MHRA for the GM1 gangliosidosis program

 Lysogene awarded Innovation Passport by the UK MHRA for the GM1 gangliosidosis program 150 150 Lysogene

First step in the recent UK MHRA initiative to accelerate the development and access to…

Lysogene secures a €4.3 million non-dilutive financing from Bpifrance to support its development

 Lysogene secures a €4.3 million non-dilutive financing from Bpifrance to support its development 150 150 Lysogene

Financing composed of a €3.0 million R&D innovation loan and a 1.3 million reimbursable advance…

Lysogene Announces a Presentation of its LYS-GM101 Program at the ESGCT 2021

 Lysogene Announces a Presentation of its LYS-GM101 Program at the ESGCT 2021 150 150 Lysogene

Paris, France — 18 October 2021 at 06:00 pm CET— Lysogene (FR0013233475 – LYS), a…

Lysogene Reports its Cash Position as of 30 September 2021

 Lysogene Reports its Cash Position as of 30 September 2021 150 150 Lysogene

Cash and cash equivalents of €9.5 million1 as of 30 September 2021 Paris, France —…

LYSOGENE Reports First Half 2021 Financial Results and Provides Operational Update

 LYSOGENE Reports First Half 2021 Financial Results and Provides Operational Update 150 150 Lysogene

In the first half 2021, Lysogene advanced and extended its development pipeline Cash and cash…

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

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

Second patient treated in the global adaptative-design clinical trial with LYS-GM101 Ongoing enrollment of a…

Lysogene Reports its Cash Position as of 30 June 2021

 Lysogene Reports its Cash Position as of 30 June 2021 150 150 Lysogene

Cash and cash equivalents of €15.0 million1 as of 30 June 2021 Paris, France —…

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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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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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