Lysogene announced that the US Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LYS-GM101, the company’s gene therapy candidate for the treatment of GM1 gangliosidosis, a serious, pediatric, life threatening disease. LYS-GM101 builds on Lysogene’s extensive experience in direct to CNS adeno-associated viral vector (AAV)-based gene therapy clinical development.
The IND clearance follows the recent clinical trial authorization granted by the MHRA in the United Kingdom. Lysogene intends to initiate its global, multi-center, single-arm, and two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis. The clinical trial will include a safety phase and a confirmatory efficacy phase. The company intends to dose a total of 16 patients, with dosage of the first patient expected in the first half of 2021.
“We are very pleased to receive this IND clearance for LYS-GM101 which completes the MHRA approval received a few weeks ago. It represents a major milestone that marks our second CNS gene-therapy program to enter into a global clinical trial” said Karen Aiach, founder chairman and chief executive officer of Lysogene. “This IND clearance once again demonstrates our quality and timely execution, and our strong determination to bring new therapeutic solutions for diseases that currently have no treatment.”
Christine Waggoner, president and co-founder of Cure GM1 Foundation added: “Children with GM1 gangliosidosis represent a clear unmet medical need and we are thrilled to see a new therapeutic option entering the clinic, as it brings tremendous hope to families and the entire GM1 gangliosidosis community.”
LYS-GM101 (‘adeno-associated viral vector serotype rh.10 expressing beta-galactosidase’) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as Rare Pediatric Disease designation in the US in 2016.