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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.
Extraordinary progress in developing therapies for CNS diseases with lead programs in neurodegenerative lysosomal storage disorders.
Our team is committed to developing pioneering therapies that will transform the lives of children affected by these devastating diseases.
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.
News, press releases, and more.
Cash and cash equivalents of €20.0 million* as of 31 March 2021 Paris, France —…
Paris, France — 13 April 2021 at 06:00pm — Lysogene (FR0013233475 – LYS), a phase…
Positive biomarker data reported with LYS-SAF302 for the treatment of MPS IIIA New program entering…
Cash and cash equivalents of €18.8 million* as of 31 December 2020 Paris, France —…
Evidence of positive biological response to LYS-SAF302 in patients treated in the AAVance clinical trial…
Second country to authorize trial initiation after recent UK MHRA approval, further country approvals pending…
Approval received from BRED for a €5 million non-dilutive financing in the form of PGE…
Paris, France — 25 January 2021 at 08:00am — Lysogene (FR0013233475 – LYS), a phase…
Paris, France — 15 January 2021 at 06:00pm — Lysogene (FR0013233475 – LYS), a phase…
LYS-GM101 is Lysogene’s second CNS gene therapy program to enter clinic Trial enrollment expected to…
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.
