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The United States Food and Drug Administration (FDA) has cleared Neurogenicis requesting a phase 1/2 clinical trial to evaluate the safety, tolerability and efficacy of NGN-101, an investigational gene therapy to treat CLN5, a form of Batten disease in late children.

The therapy uses harmless adeno-associated virus (AAV) to deliver a working copy of the CLN5 gene, which is defective in people with CLN5 Batten. Although the exact function of CLN5 is not clear, mutations in this gene lead to the accumulation of waste products inside cells, causing damage. This can trigger seizures, progressive loss of motor and intellectual abilities, visual impairment, and premature death.

“NGN-101 is the first experimental gene therapy developed to treat the devastating consequences of neurodegeneration and vision loss in patients living with Batten disease,” said Rachel McMinn, PhD, Founder and CEO of Neurogene, in a statement. Press release.

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“It is important to note that our preclinical data strongly supports the potential of NGN-101 to arrest the progression of CLN5 Batten disease, including the associated declines in vision, motor, cognition and behavior,” added McMinn.

According to the company, the trial – which is expected to begin in the first half of 2022 – will be an open-label, single-arm study to evaluate a single dose of NGN-101 given by injection into the fluid around the brain (intracerebroventricular administration) or into the eye (intravitreal administration). Neurogene also noted that the study will be the first to investigate a treatment for both neurodegeneration and vision loss in Batten.

The trial will be led by Jonathan W. Mink, MD, PhD, director of the University of Rochester Batten Center, New York, which will serve as the primary trial site in the United States.

“CLN5 is a devastating and rapidly progressive neurodegenerative disease in children that results in vision loss, cognitive and motor impairment, seizures and ultimately premature death,” Mink said. “This trial of NGN-101 will advance research into the development of a potentially disease-modifying treatment for CLN5, giving hope to individuals and families where none currently exists.”

FDA clearance was supported by preclinical data demonstrating that treatment with NGN-101 has the potential to slow or stop key features of disease progression, including vision loss, as well as declines motor, cognitive and behavioral.

NGN-101 has received orphan drug designation by the FDA and European regulatory agencies. These designations provide incentives for the development of therapies, including assistance with trial protocols, lower regulatory fees, and a period of market exclusivity if approved.

“The FDA clearance of our first [application] is an incredibly important milestone for Neurogene and brings us one step closer to our mission to bring transformative treatments to patients living with rare neurological diseases, ”concluded McMinn.

Neurogene is also conducting a natural history study (NCT03822650) of CLN5, as well as CLN7, another form of late childhood Batten disease. This study, which is still recruiting CLN5 patients, is evaluating the natural course of these diseases to determine the most important outcomes to measure in clinical trials.

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