On October 10, 2025, Neurogene Inc. announced that it had completed discussions with the U.S. Food and Drug Administration (FDA) regarding the Embolden™ registrational trial protocol for its lead gene therapy candidate, NGN-401, for Rett syndrome.
The company stated that the FDA discussions were finalized and that dosing for the Embolden trial is slated to begin in the fourth quarter of 2025, marking a key step toward advancing NGN-401 toward regulatory approval.
This regulatory update confirms that the company has secured the necessary FDA alignment on the trial design, reducing uncertainty around the pathway to approval and providing a clearer timeline for the next clinical milestones.
The announcement underscores Neurogene’s progress in translating its proprietary EXACT™ platform into a clinical program and signals momentum for the company’s rare neurological gene therapy pipeline, potentially enhancing investor confidence in the company’s development trajectory.
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