LYS-GM101 Clinical Trial
Gene therapy candidate LYS-GM101 is the subject of a Phase I/II multi-center, single-arm adaptive study in patients with infantile GM1 gangliosidosis.
Lysogene is devoted to developing
a treatment for GM1 gangliosidosis

LYS-GM101 is designed to restore the production of a key
enzyme that is missing in patients suffering from GM1
gangliosidosis, a rare inherited lysosomal storage disorder
for which there are no approved treatments.

  • LYS-GM101 is designed to restore the production of a key enzyme that is missing in patients suffering from GM1 gangliosidosis, a rare inherited lysosomal storage disorder for which there are no approved treatments. The therapy has the potential to reduce or prevent neuronal damage and thereby improve quality of life and extend the lifespan of young patients with the condition.
  • The planned safety and efficacy trial will evaluate, in an initial phase, the safety and preliminary efficacy of LYS-GM101, delivered via a one-time injection directly into the CNS (intra-cisternal administration into the cerebrospinal fluid). The second stage of this adaptive study will evaluate the efficacy of LYS-GM101 in a larger group of patients with GM1 gangliosidosis. Patients will be treated at sites in the US and Europe.

    Primary outcomes will be measured via physical and neurological examination; quality of life will also be tracked as secondary outcome. A video sub-study will collect video outcomes of patients in their home environment. This innovative, app-based approach is designed to provide further supportive evidence of the treatment effects on children’s development in a convenient and minimally invasive fashion. Video outcomes for this study will be compared to those of a Natural History Video Study initiated in Q1 2020.

  • Lysogene expects to initiate the trial in 2020. Pre-clinical work showed evidence of efficiency, good distribution of the drug candidate within the central nervous system, and a favorable risk-benefit profile.

    LYS-GM101 has obtained Orphan Drug designation in the European Union and the US, as well as Rare Pediatric Disease designation in the US.

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