Neurogene Inc. (UU8)

Overview

Neurogene Inc. is a dynamic biotechnology company focused on developing genetic medicines for patients with rare neurological diseases. The company leverages cutting-edge technology and scientific research to address the unmet needs within this challenging area of medicine. By entering into strategic license agreements with prominent institutions such as The University of North Carolina, the University of Edinburgh, Virovek, Inc., and Sigma-Aldrich Co. LLC, Neurogene Inc. has solidified its research capabilities and product development pipeline. With its headquarters in New York, New York, the company is at the forefront of genetic therapy innovations, aiming to bring transformative treatments to individuals affected by rare neurological conditions.

Products and Services

• NGN-401 for Rett Syndrome:

  NGN-401 is a leading product candidate designed for the treatment of Rett syndrome, a rare genetic neurological disorder. This innovative therapy utilizes an adeno-associated virus 9 (AAV9) as a delivery mechanism to target the root causes of the condition. Currently, NGN-401 is undergoing Phase 1/2 clinical trials, representing a significant step forward in the treatment of Rett syndrome and offering hope to those afflicted by this debilitating disease.

• NGN-101 for CLN5 Batten Disease:

  Another key product in Neurogene Inc.'s pipeline is NGN-101, aimed at treating CLN5 Batten disease, a fatal disorder of the nervous system. As a conventional gene therapy candidate, NGN-101 shows promise in modifying the disease's progression by addressing the genetic errors at their source. The therapy is in Phase 1/2 clinical trials, underscoring Neurogene's commitment to pioneering treatments for some of the most challenging neurological diseases.