Lysogene Receives Rare Pediatric Disease Designation from FDA for LYS-GM101 for Treatment of GM1 Gangliosidosis

Lysogene announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to LYS-GM101, the Company’s drug candidate in development for patients with GM1 Gangliosidosis (GM1), a severe neurodegenerative disease. The designation of Rare Pediatric Disease status makes Lysogene eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of LYS-GM101 by the FDA. LYS-GM101 has the potential to replace the defective gene in the cells of GM1 patients, which will allow for the production of the functional enzyme and prevent the progressive nature of the neurological damage caused by GM1.