Lysogene is a global biopharma leader in orphan CNS disease research and development. Lysogene has generated five non-cumulative years of clinical safety data to show the efficiency of a direct delivery route to the CNS with its initial gene therapy trial for MPS IIIA. Lysogene has recently completed the enrolment for the first multi-national observational study in MPS IIIA which will function as the non-concurrent control for the first pivotal trial for MPS IIIA in H1 2018. Lysogene also plans a clinical trial for GM1 Gangliosidosis for 2019. Lysogene has obtained orphan drug designation from the EMA and FDA and rare pediatric designation by the FDA for both programs.
Lysogene is listed on the Euronext regulated market in Paris (ISIN code: FR0013233475). For more information: www.lysogene.com