08:17 AM EDT, 10/09/2025 (MT Newswires) -- Neurogene ( NGNE ) said Thursday it has completed discussions with the US Food and Drug Administration on its Embolden registrational trial protocol and plans to initiate dosing in the fourth quarter of 2025.

The company said the trial is expected to enroll across 13 sites targeting Rett syndrome, a genetic neurological disorder that causes severe developmental regression and loss of motor and communication skills.

Neurogene ( NGNE ) also announced new preclinical data from nonhuman primates demonstrating that intracerebroventricular delivery of its NGN-401 achieves superior biodistribution across brain regions relevant to Rett syndrome, compared to intrathecal lumbar delivery.

Neurogene ( NGNE ) shares were up 2% in recent premarket activity.