Lysogene announced that the Food and Drug Administration (FDA) has granted orphan designation to LYS-GM101, their gene therapy drug candidate for the treatment of GM1 Gangliosidosis (GM1).

GM1 is an extremely severe, inherited disorder that progressively destroys neurons in the brain and spinal cord. It is caused by a mutation in the GLB1 gene encoding for the lysosomal acid beta-galactosidase (ßgal) enzyme.

LYS-GM101 is designed to replace a gene mutation in the cells in order to allow for production of the functional enzyme and to prevent the progressive nature of neurological damage caused by GM1.

“It is good news for patients that we can further study this therapy as this is a severe neurodegenerative disease with unmet medical need,” said CEO of Lysogene, Karen Aiach, about the FDA’s decision. “We look forward to starting our upcoming phase I/II clinical trial (LYS-GM101) by the end of the first half of 2018, in line with the timetable previously announced.”

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