Lysogene Announces Positive DSMB Findings Recommending Study Continuation and Positive Biomarker Data in Phase 1 of Adaptive Gene Therapy Clinical Trial with LYS-GM101 for the Treatment of GM1 Gangliosidosis – 11/29/ 2022 at 6:00 PM


• Finalization of the treatment and follow-up at 6 months of the last patient recruited in the first phase of the adaptive clinical trial P1-GM-101

• The DSMB gave a favorable opinion for the continuation of the study after the review of the safety data for the 5 subjects treated with LYS-GM101, paving the way for the initiation of the second phase

• Positive biomarker data demonstrating long-lasting biological activity of LYS-GM101

Paris, France — November 29, 2022 at 6:00 p.m. CET — Lysogene (FR0013233475 – LYS), a Phase 3 biopharmaceutical company based on a gene therapy technology platform targeting diseases of the central nervous system (CNS), announces today that the Independent Data Safety Monitoring Board (DSMB) has completed a review of the safety data from the first phase of the adaptive clinical trial with the investigational gene therapy LYS-GM101, an AAVrh10 vector containing the coding sequence of beta -human galactosidase (β-gal), for the treatment of GM1 gangliosidosis and recommended continuation of the study as planned.



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