PARIS: Lysogene, a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, has received MHRA and Research Ethics Committee approvals to initiate the gene therapy clinical trial in the United Kingdom with LYS-GM101 for the treatment of GM1 gangliosidosis, a serious, pediatric, life threatening disease.

LYS-GM101 builds on Lysogene’s extensive experience in direct to CNS adeno-associated viral vector (AAV)-based gene therapy clinical development.

This is the first regulatory authorization to initiate this global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis. The company expects to dose the first patient in the first half of 2021.

“This trial builds on Lysogene’s considerable expertise for AAV gene therapy and clinical development. Since the most damaging effects of GM1 gangliosidosis occur in the brain and spinal cord, LYS-GM101 will be administered into a cerebrospinal fluid-filled space at the back of the head called the cisterna magna so that enough therapy reaches those tissues. I expect the study to provide us with data demonstrating the transformative potential of LYS-GM101” said Karen Aiach, Founder Chairman and Chief Executive Officer of Lysogene.

“LYS-GM101 is our second gene therapy program entering the clinical phase after LYS-SAF302 for MPS IIIA, followed by a discovery stage pipeline, including gene therapy candidates for Fragile X syndrome, Gaucher and Parkinson’s disease.”

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.

Leading international gene therapy and Lysosomal Storage Disease centers plan to participate in the clinical trial (NCT04273269).

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 in infantile and juvenile GM1 gangliosidosis (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 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. www.lysogene.com

Manchester University NHS Foundation Trust is one of the largest NHS trusts in England and a leading provider of specialist healthcare services. Its nine hospitals are home to hundreds of world class clinicians and academic staff committed to finding patients the best care and treatments. www.mft.nhs.uk