Lysogene Announces First Patient Receiving Experimental LYS-GM101 Gene Therapy for Treatment of GM1 Gangliosidosis
PARIS–(COMMERCIAL THREAD) – Regulatory news:
Lysogen (Paris: LYS) (FR0013233475 – LYS), a Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced the dosage of the first patient, at the Royal Manchester Children’s Hospital, part of the Manchester University NHS Foundation Trust , in the global adaptive design clinical trial with LYS-GM101 (NCT04273269), a gene therapy for the treatment of GM1 gangliosidosis.
This trial is an interventional, multicenter, single-arm, adaptively designed, two-step study evaluating the intracisternal administration of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human gene for β-galactosidase (GBL1). The clinical trial includes a safety phase and a confirmation of efficacy phase. The trial will enroll 16 patients diagnosed with early or late childhood GM1 gangliosidosis at sites in the United States and Europe. More information can be found at www.clinicaltrials.gov.
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 to date for this disease.
“Dosing the first patient in this clinical study is an important milestone for Lysogene, as it marks the second gene therapy program in our portfolio to enter the clinic. It once again illustrates our ability to perform despite the recent challenges we have faced with Covid-19 “ mentionned Karen Aiach, Founding President and CEO of Lysogene. “We are always looking for opportunities to accelerate the development of new gene therapy treatments to improve the lives of patients and are excited to bring this experimental therapy to patients with GM1 gangliosidosis.
“GM1 gangliosidosis is a devastating and rapidly progressive disease, particularly in its most severe form where it causes profound neurodegeneration and early death”, mentionned Simon Jones, MD, chief researcher and consultant in pediatric hereditary metabolic diseases at the Royal Manchester Children’s Hospital, part of the 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 the United States in 2017, as well as the rare pediatric disease designation in the United States in 2016.
Lysogene is also funding a natural history study of GM1 gangliosidosis conducted by Casimir Trials to collect prospective and / or retrospective videos of children performing certain daily tasks and behaviors (NCT04310163).
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 the safe and effective delivery of CNS gene therapies 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 underway 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 the exclusive commercial rights for LYS-SAF302 in the United States and in markets outside Europe; and Lysogene will retain the commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the development strategy for the treatment of fragile X syndrome, a genetic disease linked to autism. www.lysogene.com.
About Manchester University NHS Foundation Trust
The Manchester University NHS Foundation Trust is one of the largest NHS Trusts in the country and a leading provider of specialist health services. Its 10 hospitals are home to hundreds of world-class clinicians and academic staff, committed to finding the best care and treatment for patients.
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 www.mft.nhs.uk
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