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

Lysogene (Paris:LYS) (FR0013233475 – LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced dosing of the first patient, at Royal Manchester Children’s Hospital, part of Manchester University NHS Foundation Trust, in the global adaptative-design clinical trial with LYS-GM101 (NCT04273269), a gene therapy for the treatment of GM1 gangliosidosis.

This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene (GBL1). The clinical trial includes a safety phase and a confirmatory efficacy phase. The trial will enroll 16 patients with a diagnosis of early or late infantile GM1 gangliosidosis at sites in the US and Europe. More information can be found on

GM1 gangliosidosis is a fatal autosomal recessive disease caused by mutations in the GLB1 gene leading to accumulation of GM1 ganglioside in neurons resulting in progressive neurodegeneration. No treatment has been approved so far for this disease.

“Dosing the first patient in this clinical study represents a significant milestone for Lysogene, as it marks the second gene therapy program from our portfolio to enter the clinic. It illustrates once again our ability to execute despite the recent challenges we faced with Covid-19” said Karen Aiach, Founder Chairman and Chief Executive Officer of Lysogene. “We are always looking for opportunities to accelerate the development of new gene therapy treatments to improve patients’ lives and are excited to bring this investigational therapy to patients with GM1 gangliosidosis.”

“GM1 gangliosidosis is a devastating and rapidly progressing disease, especially in its most severe form where it causes profound neurodegeneration and early death”, said Simon Jones, M.D., Chief Investigator and Consultant in Paediatric Inherited Metabolic Disease at Royal Manchester Children’s Hospital, part of Manchester University NHS Foundation Trust (MFT) and Senior Lecturer at The University of Manchester. “I am delighted to have started injecting the first patient with LYS-GM101, whose administration went perfectly well”.

LYS-GM101 (‘adeno-associated viral vector serotype rh.10 expressing beta-galactosidase’) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as the Rare Pediatric Disease designation in the US in 2016.

Lysogene is also funding a GM1 gangliosidosis natural history study being conducted by Casimir Trials to collect prospective and/or retrospective videos of children doing certain everyday tasks and behaviors (NCT04310163).

About Lysogene
Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/3 clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism.

About Manchester University NHS Foundation Trust
Manchester University NHS Foundation Trust is one of the largest NHS Trusts in the country and a leading provider of specialist healthcare services. Its 10 hospitals are home to hundreds of world class clinicians and academic staff, committed to finding patients the best care and treatments.
Its hospitals are Manchester Royal Infirmary, Saint Mary’s Hospital, Royal Manchester Children’s Hospital, Manchester Royal Eye Hospital, University Dental Hospital of Manchester, Trafford General, Altrincham Hospital, Wythenshawe Hospital, Withington Hospital and North Manchester General Hospital.
More information is available at

Stéphane Durant des Aulnois
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