Lysogene uses a recombinant adeno-associated virus (AAV) that specifically targets neural cells and is administered directly into the brain or cerebrospinal fluid (CSF). This direct-to-CNS approach has several advantages. The therapy does not have to cross the blood-brain-barrier, and as a consequence more of the drug reaches the intended site of action. Relatively low doses can therefore be used, helping reduce local and systemic side-effects. Finally, because the therapy is delivered directly to the CNS, even patients with pre-existing immunity to the AAV capsid, who cannot be treated by systemically administered AAV, can benefit from the treatment.

In the case of neuronopathic LSDs, correcting only a small subset of neural cells using gene therapy may be sufficient to reduce storage of cells across much larger regions of the CNS. That’s because some lysosomal enzymes can be secreted from the cells in which they are produced and be taken up and used by other neighboring cells. 

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.

This technology platform is applicable to other central nervous system diseases. Lysogene’s goal is to expand our therapeutic capabilities into next-generation gene therapy technologies to help and heal those affected by rare diseases.