Lysogene announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to LYS-GM101, the company’s gene therapy drug candidate for treatment of GM1 Gangliosidosis (“GM1”).
“FDA Orphan Drug Designation for LYS-GM101 is an important regulatory milestone, which will facilitate clinical development of LYS-GM101. It is good news for patients that we can further study this therapy as this is a severe neurodegenerative disease with unmet medical need,” stated Karen Aiach, Founder and Chief Executive Officer of Lysogene. “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.”
LYS-GM101 is designed to replace a defective gene in the cells of GM1 patients, in order to allow for production of the functional enzyme and to prevent the progressive nature of the neurological damage caused by GM1 in humans.