Northwest Rett Syndrome Association

Today, Neurogene Inc. announced the completion of dosing in the Embolden registrational trial of investigational gene therapy, NGN-401.

Due to strong demand, 25 participants have been dosed, exceeding the original target of 20, and NGN-401 has been generally well tolerated, with no cases of HLH at the 1E15 vg dose. Neurogene expects to share updated interim Phase 1/2 data in mid-2026 and topline data from Embolden in the second half of 2027.

We look forward to welcoming the Neurogene team to the IRSF Rett Syndrome Scientific Meeting and ASCEND National Summit later this month.

Read the full press release here: https://ir.neurogene.com/news-releases/news-release-details/neurogene-announces-successful-completion-dosing-emboldentm