Neurogene Initiates Natural History Study of Charcot-Marie Tooth Disease (CMT4J)

Key data now being collected to support endpoint assessment for future gene therapy clinical trial

NEW YORK–(BUSINESS WIRE)–Neurogene Inc., a company founded with a mission to bring life-changing medicines to patients and families affected by rare neurological diseases, today announced that it has enrolled the first patient in a natural history study of Charcot-Marie Tooth disease, type 4 (CMT4J), a rare inherited peripheral neuropathy. The goal of the study is to provide important data to support a future gene therapy clinical trial. This natural history study is being conducted under a collaboration between Neurogene and UT Southwestern Medical Center as one of multiple sponsored research agreements.

Charcot-Marie Tooth disease is a group of the most common inherited peripheral and sensory neuropathies caused by pathogenic changes in genes that affect peripheral nerve axons or the myelin sheath. CMT4J is a rare form of CMT caused by defects in the FIG4 gene that lead to uneven loss of myelin in sensory and motor nerve axons. Individuals with CMT4J typically develop symptoms of weakness and muscle atrophy in the upper and lower extremities in childhood. Early onset of the disease can be associated with a rapidly progressive course, ultimately leading to loss of ambulation, quadriplegia, respiratory compromise and premature death. Later disease onset has a more variable course that can lead to transient muscle weakness for some patients and rapid onset of symptoms for others.

“This is the world’s first prospectively designed natural history study specifically for CMT4J,” said Diana Castro, M.D., Assistant Professor at UT Southwestern Medical Center and lead investigator of the study. “With the help of the patients participating, this work will generate invaluable data to help us define optimal endpoints for a future planned gene therapy trial.”

“Patients with CMT4J and their families have been waiting for a therapy, and we view this study as a critical component of the clinical development plan to address that unmet need,” said Effie Albanis, M.D., Neurogene’s Chief Medical Officer. “We are taking a streamlined approach to clinical development to facilitate the fastest possible approval timeline for a future gene therapy medicine, consistent with our belief that patients should not be waiting longer than necessary for life-altering medicines.”