Neurogene Shares Rise After NGN-401 Discussions With FDA
By Chris Wack
Neurogene shares were 11% higher, at $28.70, after the company said it has completed discussions with the Food and Drug Administration on its Embolden registrational trial protocol for its NGN-401 gene therapy for Rett Syndrome.
The company said it plans to initiate dosing in the fourth quarter of 2025, and the trial is expected to enroll across 13 sites.
Neurogene said it is well-positioned to engage in early discussions with the FDA with the goal of an expedited BLA submission.
Neurogene also said it saw positive preclinical data from nonhuman primates demonstrating that intracerebroventricular delivery of NGN-401 achieves superior adeno-associated viru biodistribution across brain regions relevant to Rett syndrome, compared to intrathecal lumbar delivery.
These findings are consistent with preclinical data generated by other independent laboratories, reinforcing a growing body of evidence that supports ICV delivery for achieving greater expression in the brain.
Write to Chris Wack at chris.wack@wsj.com
(END) Dow Jones Newswires
October 09, 2025 10:24 ET (14:24 GMT)
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