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 it is conducting a natural history study of CLN7 and CLN5, two forms of late-infantile variant Batten disease. The goal of the study is to better understand the course of the disease and to determine clinically meaningful outcome measures for use in future gene therapy trials. This natural history study is being conducted under a collaboration between Neurogene and UT Southwestern Medical Center as one of multiple sponsored research agreements.
Batten disease, also called neuronal ceroid lipofuscinoses (NCL), is a family of rare and fatal neurodegenerative diseases caused by pathogenic changes in one of a series of genes that result in the accumulation of toxic deposits across multiple organ systems, including the brain, eye, skin and other cells. CLN7 and CLN5 are rare, pediatric-onset and rapidly progressive diseases caused by defects in the CLN7 and CLN5 genes, respectively.
CLN7 or CLN5 diseases are characterized by seizures, progressive deterioration in intellectual and motor capabilities, loss of vision and death in childhood or adolescence. Diagnosis of the disease is confirmed through genetic testing.
“CLN7 and CLN5 are devastating and severe neurological diseases, and there are currently no treatments for them,” said Berge Minassian, M.D., Division Chief of Child Neurology at UT Southwestern Medical Center. “Through this study, and thanks to those in the Batten disease community who choose to participate, we will soon know much more about how the disease affects those who suffer from it so that we may help the many children in need.”
“Neurogene is committed to reimagining the future for people living with rare neurological diseases,” said Effie Albanis, M.D., Neurogene’s Chief Medical Officer. “This natural history study of CLN7 and CLN5 will be instrumental in designing a gene therapy to treat these rare forms of Batten disease.”
About the natural history study
Neurogene is sponsoring a prospective, observational natural history and outcome measure discovery study into which approximately 30 patients with genetically confirmed CLN7 or CLN5 will be enrolled. Patients currently being enrolled at UT Southwestern Medical Center will undergo multiple assessments, including electroencephalography, or EEG tests, neuropsychological and ophthalmological evaluations and annual MRI scans of the brain, among others. Saima Kayani, M.D., Assistant Professor at UT Southwestern Medical Center, is the lead investigator. For additional information about the trial, please visit www.clinicaltrials.gov, NCT03822650.
About Neurogene Inc.
Neurogene was founded to bring life-changing medicines to patients and families affected by rare neurological disorders. We partner with leading academic researchers, patient advocacy organizations and caregivers to bring to patients therapies that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options exist today. Our lead programs 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 www.neurogene.com