Lysogene’s gene therapy procedure is safe and well-established. Administration occurs during a single medical procedure. The injection locations are specific to each of the diseases we target.
For MPS IIIA, a disease that primarily affects the brain, our therapeutic candidate LYS-SAF302 is administered by direct intracerebral infusion. For GM1 gangliosidosis, affecting both the brain and the spinal cord, our therapeutic candidate LYS-GM101 is administered via intra-cisternal injection into the cerebrospinal fluid.