lixmabegagene relduparvovec

Lixmabegagene relduparvovec is an investigational gene therapy developed for the treatment of GM1 gangliosidosis, a rare inherited neurodegenerative disorder. It utilizes a recombinant adeno-associated virus serotype rh.10 (AAVrh.10) vector to deliver the human GLB1 gene, which encodes the enzyme beta-galactosidase. This enzyme is essential for recycling GM1-ganglioside in neurons; its deficiency leads to progressive destruction of nerve cells in the brain and spinal cord. The therapy aims to restore beta-galactosidase activity through gene transference and replacement, potentially addressing the underlying cause of GM1 gangliosidosis[1][3][4].