Develops genetic medicines for neurological diseases, focusing on gene therapies and disease-modifying treatments.
Neurogene Inc. is a pioneering biotechnology firm dedicated to advancing genetic medicines tailored for rare neurological disorders. Leveraging cutting-edge research, the company focuses on developing innovative therapies that address significant unmet medical needs within this specialized field.
Among its promising product candidates is NGN-401, currently undergoing Phase 1/2 clinical trials. Packaged in an adeno-associated virus 9 vector, NGN-401 aims to provide a potential treatment for Rett syndrome, a rare genetic disorder affecting the nervous system. Additionally, Neurogene's pipeline includes NGN-101, an advanced gene therapy in Phase 1/2 trials targeting CLN5 Batten disease, a devastating neurodegenerative condition.
Neurogene Inc. has established strategic partnerships through licensing agreements with prominent institutions such as The University of North Carolina, the University of Edinburgh, Virovek, Inc., and Sigma-Aldrich Co. LLC. These collaborations bolster the company's capabilities in genetic medicine development and underline its commitment to advancing therapeutic options for patients. Headquartered in New York, New York, Neurogene continues to drive innovation in rare neurological disease treatments, aiming to transform the lives of patients and families worldwide.