NEW YORK–(COMMERCIAL THREAD) –Neurogen Inc., a company founded with a mission to bring life-changing drugs to patients and families affected by rare neurological diseases, today announced that it is conducting a natural history study of CLN7 and CLN5, both forms of the late infantile variant of Batten disease. The aim 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 as part of a collaboration between Neurogene and UT Southwestern Medical Center under several sponsored research agreements.
Batten disease, also known as neuronal ceroid lipofuscinosis (NCL), is a family of rare and fatal neurodegenerative diseases caused by pathogenic changes in one of a series of genes that cause toxic deposits to build up in multiple systems of ‘organs, including the brain, eyes, skin and other cells. CLN7 and CLN5 are rare, pediatric-onset, rapidly-progressive diseases caused by defects in the CLN7 and CLN5 genes, respectively.
CLN7 or CLN5 diseases are characterized by seizures, progressive deterioration of intellectual and motor skills, loss of vision, and death during childhood or adolescence. The diagnosis of the disease is confirmed by genetic tests.
“CLN7 and CLN5 are devastating and serious neurological diseases, and there is currently no cure for them, ”said Berge Minassian, MD, division chief of childhood neurology at UT Southwestern Medical Center. “Through this study, and thanks to those in the Batten Disease community who have chosen to participate, we will soon know a lot more about how the disease affects those who have it so that we can help the many children in the world. need.
“Neurogene is committed to reinventing the future of people living with rare neurological diseases, ”said Effie Albanis, MD, Medical Director of Neurogene. “This natural history study of CLN7 and CLN5 will be instrumental in the design of gene therapy to treat these rare forms of Batten disease.
About the study of natural history
Neurogene is sponsoring a prospective, observational, natural history discovery and outcome measurement study in which approximately 30 patients with genetically confirmed CLN7 or CLN5 will be enrolled. Patients currently enrolled at UT Southwestern Medical Center will undergo multiple assessments, including electroencephalography or EEG tests, neuropsychological and ophthalmological assessments, and annual brain MRIs, among others. Saima Kayani, MD, assistant professor at UT Southwestern Medical Center, is the principal investigator. For more information on the trial, please visit www.clinicaltrials.gov, NCT03822650.
About Neurogene Inc.
Neurogene was founded to provide life changing drugs for patients and families affected by rare neurological disorders. We work in partnership with leading academic researchers, patient advocacy organizations and caregivers to provide patients with therapies that address the underlying genetic cause of a wide range of neurological diseases for which no options are available. effective treatment does not exist today. Our main programs use AAV-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also investing in new technology to develop treatments for diseases underserved by gene therapy. For more information visit www.neurogene.com