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Completed discussions with FDA on registrational protocol; 13 sites to allow for rapid enrollment
New preclinical data at ESGCT Congress highlight NGN-401 best-in-class potential and add to body of evidence that ICV administration is superior to IT-L in reaching key brain regions underlying Rett syndrome
As previously reported, the first four participants in the NGN-401 Phase 1/2 trial gained 23 developmental milestones/skills, representing multi-domain improvement that requires transduction of key brain regions and nervous system
The preclinical data also provide evidence-based confirmation that ICV and IT-L administration result in comparable peripheral organ transduction, showing no liver-sparing benefit to IT-L when compared to ICV
Neurogene Inc. (NASDAQ:NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the Company has completed discussions with the U.S. Food and Drug Administration (FDA) on its Embolden™ registrational trial protocol and plans to initiate dosing in the fourth quarter of 2025. In response to strong interest from the Rett syndrome community, the trial is expected to enroll across 13 sites.
Posted In: NGNE
