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Neurogene Announces First Patient Enrolled in Natural History Study Evaluating Two Subtypes of Batten Disease

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Study Enrolling Patients with CLN5 and CLN7 Subtypes Aims to Help Understand Natural Disease Course and to Help Identify Potential Clinical Endpoints for Future Gene Therapy Studies

NEW YORK, November 12, 2020 – Neurogene Inc., a company founded with a mission to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announces the first patient is enrolled in a natural history study of CLN5 and CLN7, two subtypes of late-infantile variant Batten disease. The study is being conducted at the University of Rochester Batten Disease Center.

The study will evaluate patients to assess the course of the disease and may aid in determining potential clinical trial endpoints for use in future gene therapy clinical trials treating patients with CLN5 or CLN7 subtypes. In response to the COVID-19 pandemic, and to expand the number of families able to participate, all patients will be able to enroll and to participate in the study either in person or through telemedicine visits. Importantly, patients who participate in this Natural History Study may be considered for enrollment in future Neurogene gene therapy trials, based on meeting inclusion and exclusion criteria.

“As parents, we know that any new research on Batten disease, particularly on CLN5 and CLN7 subtypes, could help improve care for children with this difficult condition, including our daughter,” said Hemaxi and Rishi Patel, whose child is living with CLN7. “That is why we are so thankful the study is now allowing us and many others to participate virtually from home to help doctors learn more about this rare disease which, hopefully, will lead to potential new treatments.”

“Today’s announcement is an important milestone for patients, families and Neurogene,” said Rachel McMinn, Ph.D., Neurogene Founder and Chief Executive Officer. “This study is designed to provide critical insights into Batten disease, including identifying important clinical endpoints and enabling researchers to gain a better understanding of this disease.”

About the Natural History Study
Neurogene is sponsoring a prospective, natural history and outcome measure discovery study, enrolling approximately 30 patients with genetically confirmed CLN5 or CLN7. Families and patients will be able to participate in the study visits either in person or remotely, to perform assessments and complete questionnaires. These assessments may include the Unified Batten Disease Rating Scale (UBDRS), Late Infantile Neuronal Ceroid Lipofuscinosis Rating Scale (Hamburg Scale), and Vineland Adaptive Behavior Scale (II or 3), among others. Lead investigator for the study is Jonathan W. Mink, M.D., Ph.D., Director of the University of Rochester Batten Center. Dr. Mink also is the Frederick A. Horner, M.D. Professor in Pediatric Neurology and Professor of Neurology, Neuroscience, and Pediatrics at the University of Rochester, where he is also Chief of Child Neurology. For additional information or questions about the trial, please visit www.clinicaltrials.gov, NCT03822650, medicalinfo@neurogene.com or by calling 1-866-623-5093.

About Batten disease
Batten disease, also called neuronal ceroid lipofuscinoses (NCL), is a family of rare neurodegenerative diseases caused by pathogenic changes in one of a series of genes that results in the accumulation of toxic deposits across multiple organ systems, including the brain, eye, skin and others. CLN5 and CLN7 are rare, pediatric-onset and rapidly progressive subtypes caused by a variant in the CLN5 and MFSD8 genes, respectively. They are characterized by seizures, progressive decline in intellectual and motor capabilities, and loss of vision in childhood or adolescence. Diagnosis of the disease is confirmed through genetic testing. Neurogene has developed an educational website, www.LivingBatten.com to support patients, families and care providers looking for more information on CLN5 and CLN7 subtypes of Batten disease.

About Genetic Testing
Neurogene is committed to lowering the barriers of obtaining a genetic diagnosis for patients and has partnered with Invitae to co-sponsor two genetic testing programs in the US and in Canada. Healthcare providers can order, at no cost, an Invitae Epilepsy panel for any child under the age of eight who has had an unprovoked seizure. The Detect Lysosomal Storage Diseases panel is available at no charge to patients who are suspected of having a lysosomal storage disease. Visit https://www.invitae.com/en/sponsored-testing/ for more details.

About Neurogene Inc.
Neurogene Inc. is focused on developing life-changing genetic medicines for patients and their families affected by rare, devastating neurological diseases. We partner with leading academic researchers, patient advocacy organizations, and caregivers to bring therapies to patients that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options exist today. Our lead programs are designed to use AAV-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also investing in novel technology to develop treatments for diseases not well served by gene therapy. For more information, visit neurogenestg.wpengine.com.

Media Contact
Linda Phelan Dyson, MPH

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