There are no effective therapies for the vast number of RARE NEUROLOGICAL DISEASES

“My passion for wanting to find a treatment for devastating neurological diseases dates back to me as a child, living with an older brother severely affected by autism. Autism wasn’t a household word in those days, and his disease profoundly impacted me and my entire family. When I learned about the benefits of gene therapy in a different devastating neurological disorder, I was convinced gene therapy would become a viable tool with the potential to provide profound treatment benefits. I felt an obligation to start Neurogene in order to help ameliorate the suffering in other families going through a similar experience to my own.”

– Rachel McMinn, Ph.D., CEO

Neurogene is accelerating development of new genetic medicines to people with devastating neurological diseases and their families. To do this, we are working with experts across the globe to advance a broad pipeline of programs to treat the underlying cause of serious neurological disorders and thereby address the overwhelming need for new therapies.

Although the human genome was sequenced for the first time nearly 20 years ago, the true genomics revolution is taking place now, providing us with the ability to radically alter rare, genetic disorders. At Neurogene, we are working to provide medicines to improve the lives of neurologically-impaired and developmentally-delayed children and their families.

For children, our goal is simple: to enable them to have a healthier future. For their families, we want to make their lives better: when one family member is neurologically impaired, there is a heavy burden on caregivers – whether it is caring for an infant who will not live to see her third birthday, communicating with a son who doesn’t speak, or attempting to control a teenager in the throes of a self-injurious tantrum.


Neurogene | Our company's mission. Neurogene | Our company's mission.