Neurogene Completes Enrollment in Embolden Trial of NGN-401

MT Newswires Live
Jun 08

Neurogene (NGNE) has completed dosing in the Embolden registrational trial of NGN-401 for Rett syndrome, exceeding its initial target by enrolling 25 participants, the company said Monday.

Neurogene said the trial was overenrolled due to strong demand from the Rett syndrome community and that NGN-401 at the 1E15 vg dose has been generally well tolerated, with no cases of hemophagocytic

lymphohistiocytosis reported.

The company said topline data from the Embolden trial are expected in the second half of next year.

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