Research & Development
US FDA Clears IND for Neurogene's NGN-101 Gene Therapy to Treat CLN5 Batten Disease
15 September 2021 - - The US Food and Drug Administration has cleared US-based rare neurological disease therapeutics developer Neurogene Inc's Investigational New Drug application for NGN-101, the company said.

NGN-101, Neurogene's lead product candidate in its Batten disease franchise, is an investigational adeno-associated virus gene therapy for the treatment of CLN5 Batten disease, a rare and fatal neurodegenerative disorder with no approved disease-modifying therapies available.

IND clearance enables Neurogene to initiate a Phase 1/2 trial to assess the safety, tolerability, and efficacy of NGN-101 in patients with CLN5 Batten disease.

The planned open-label, single-arm trial will evaluate a single dose delivered via intracerebroventricular and intravitreal 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., the Frederick A. Horner MD Distinguished Professor in Pediatric Neurology, Chief of Child Neurology, and director of the University of Rochester Batten Center.

NGN-101 is being developed as a one-time treatment via intracerebroventricular and intravitreal routes of administration for CLN5 Batten disease using an adeno-associated virus to deliver the gene encoding CLN5, which is deficient in children with CLN5 Batten disease.

Preclinical data strongly suggest that NGN-101 treatment has the potential to halt the key features of disease progression in patients with CLN5 Batten disease, including the associated vision, motor, cognitive, and behavioral declines. NGN-101 has received Orphan Drug Designation by the US and European regulatory agencies.

Batten disease is a group of rare, inherited neurodegenerative diseases also called neuronal ceroid lipofuscinoses, in which a pathogenic genetic change in one of 13 different genes results in the accumulation of toxic deposits across multiple organ systems, culminating in progressive deterioration in intellectual and motor capabilities, loss of vision, and premature death.

The different subtypes of Batten disease share many common features, and a diagnosis requires genetic testing to confirm the cause of disease.

CLN5 Batten disease is caused by a pathogenic variant in the CLN5 gene, and is characterized by loss of vision, seizures, and progressive decline in intellectual and motor capabilities with initial signs and symptoms starting in childhood.

Neurogene has developed an educational website,, to support patients, families, and care providers looking for more information, resources, and genetic testing.

The company's lead programs use adeno-associated virus vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene.

Neurogene is also developing novel gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully address.


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