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Neurogene Inc. (NYC, USA) has announced to become the first investigative treatment of both neurodegenerative and ocular disease manifestations of Batten Disease.

Neurogene Inc. based in Manhattan, New York City, has announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for “NGN-101”, an investigational treatment of CLN5 Batten disease, a rare and fatal neurodegenerative disorder with no approved disease-modifying therapies.  According to the company, the NGN-101 strategy is being developed as a one-time treatment via intracerebroventricular (ICV) and intravitreal (IVT) routes of administration for CLN5 Batten disease.  Last year, Neurogene Inc. raised $115 million in a second-round financing to be used to accelerate clinical trials with the financing round was led by EcoR1 Capital.


Batten disease is a common name for a group of disorders based on neuronal ceroid lipofuscinoses (NCL).  At least 13 genes, primarily autosomal recessive, have been reported in association with the disease, and the most prevalent form of Batten disease has been linked to mutations in the CLN3 gene (lysosomal/endosomal transmembrane gene) on chromosome 16, encoded by battenin, a transmembrane protein first described in 1903. The worldwide prevalence of Batten disease is ~1 in 100,000 live births and until the past few years there is no effective treatment for this condition. Retinal disease starts with macular involvement (red-cherry spot) and later expands to peripheral retina. The protein encoded by CLN3 is found in the lysosomes and in synapses and this can build up in nervous tissue, causing seizures, visual impairment, mobility loss and early death.  The general diagnosis of Batten disease is based on a combination of clinical signs and symptoms, ophthalmological evaluations, EEG and brain MRI, and is subsequently confirmed with genetic and biochemical tests. There are up to eleven (11) NCL sub-types (CLN1-11) and NGN-101, directed to CLN5, is caused by a pathogenic variant characterized by loss of vision, seizures, and progressive decline in intellectual and motor capabilities with initial signs and symptoms, starting in childhood. In the current Phase 1/2 clinical trial, the study enables the company to initiate the safety, tolerability, and efficacy of NGN-101 in patients. The planned open-label, single-arm trial will evaluate a single dose delivered via intracerebroventricular (ICV) and intravitreal (IVT) routes of administration. University of Rochester will serve as the lead trial site in the United States, led by Jonathan W. Mink, M.D., Ph.D., Professor in Paediatric Neurology and Director of the University of Rochester Batten Centre.


According to the company’s chief executive and founder, Rachel McMinn, Ph.D., stated, “NGN-101 is the first investigational gene therapy developed to address the devastating consequences of both neurodegeneration and vision loss in patients living with Batten disease. Importantly, we believe our preclinical data strongly support the potential of NGN-101 to stop the progression of CLN5 Batten disease, including the associated vision, motor, cognitive, and behavioral declines. FDA clearance of our first IND is an incredibly significant milestone for Neurogene, and moves us one step closer to delivering on our mission to bring transformative treatments to patients living with rare neurologic diseases.”