08 Mar
NIB is honored to support development of Lysogen's LYS-GM101 gene therapy

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NIB is honored to support development of Lysogen's LYS-GM101 gene therapy

On February 12, Lysogene received FDAs clearance of Investigational New Drug (IND) Application to initiate the gene therapy clinical trial in the US with LYS-GM101 for the treatment of GM1 Gangliosidosis (https://bit.ly/3kRLCsH).

LYS-GM101 is the Lysogene’s gene therapy candidate for the treatment of GM1 gangliosidosis, a serious, pediatric, life threatening disease.

National institute of Biology (NIB) serves as a contract lab for Lysogene, providing time-effective support with specific analytical techniques, such as droplet digital polymerase chain reaction (ddPCR), transmission electron microscopy (TEM) and High-Throughput Sequencing (HTS), required for characterization of viral vectors.

NIB is honoured to be a part of this milestone and is grateful for trust received by Lysogene.

About Lysogene
Lysogene (www.lysogene.com) 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 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. An adaptative 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 NIB
Department of Biotechnology and Systems Biology at the National Institute of Biology is holder of worldwide recognized expertise in the field of virus diagnostics, characterization and quantification.