Neurogene and University of Edinburgh Announce Research Collaboration to Advance Next Generation Gene Therapies

Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, and University of Edinburgh, a world leader in biomedical and translational research for neurodevelopmental diseases, today announced a research collaboration to advance development of multiple platform approaches to enable next generation gene therapies.

The collaboration provides comprehensive research capabilities to Neurogene, enabling the company and the University to expedite a multiple platform approach to improve upon existing gene therapy technologies. Under the terms of the collaboration, Neurogene will provide financial support for Dr. Stuart Cobb's laboratory at the University of Edinburgh, in exchange for the right to license any applicable intellectual property at agreed-upon economic terms. Neurogene will be responsible for late stage preclinical and all clinical development of any products generated under the collaboration.

"This partnership provides Neurogene with preeminent neurological research expertise and capabilities. Dr. Cobb's lab has contributed significant scientific expertise to improve the quality of our current rare disease pipeline and generated promising early data to allow us to tackle complex neurological diseases not addressable with conventional gene therapy," said Rachel McMinn, Ph.D., Founder and CEO of Neurogene. "With this collaboration, I look forward to advancing our mission to provide safe and effective genetic therapies to patients and families as quickly as possible."

"Neurogene is a science-driven company committed to investing in innovation and is the right partner for us to build upon the early successes in gene therapy technology," Stuart Cobb, Ph.D., Simons Fellow and Reader in Neuroscience at the Patrick Wild Centre and Centre for Discovery Brain Sciences, University of Edinburgh, stated. "We are excited to collaborate with Neurogene on the critically-important endeavor of improving upon current gene therapy technologies. Gene therapy is a very promising yet complex development area, and we are privileged to help address the unmet needs that exist within rare neurological diseases."

In addition to Dr. Cobb's position at the University of Edinburgh, he serves as Chief Scientific Officer of Neurogene.

This Collaboration has been supported by Edinburgh Innovations, the University of Edinburgh's commercialization service.

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 currently exist. Our lead programs use adeno-associated virus (AAV) 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. For more information, visit www.neurogene.com.