Neurogene Announces Trial Design for Embolden™ Study of NGN-401 Gene Therapy for Rett Syndrome

Neurogene has announced key design elements of its upcoming registrational clinical trial, Embolden™, for NGN-401, an investigational gene therapy for Rett syndrome. A registrational clinical trial gathers a comprehensive set of data for review by regulatory agencies like the FDA or EMA with the goal of obtaining regulatory approval to market the therapeutic. 

Here are some highlights:

• The trial will include females aged 3 and older with a total of 18 patients expected to be enrolled.
• Participants will receive a single dose of NGN-401 in an open-label, single-arm, baseline-controlled study.
• The primary efficacy endpoint will assess improvement over a 12 month period using the CGI-Improvement scale, along with the gain of at least one developmental milestone or skill from a list of 28.

Trial site locations have not yet been determined, and enrollment is not yet open. Neurogene plans to provide updates as preparations continue and will post more information once enrollment begins.

You can read more in the materials released by Neurogene below.