Neurogene Completes Dosing in Registrational Trial of NGN-401 Gene Therapy for Rett Syndrome; Topline Data Expected 2H 2027

Neurogene Inc. has completed dosing in its Embolden registrational trial of NGN-401, a gene therapy for Rett syndrome, exceeding the target by dosing 25 participants due to strong demand from the patient community. The therapy has been generally well-tolerated with no cases of HLH (hemophagocytic lymphohistiocytosis) at the 1E15 vg dose. Additional interim Phase 1/2 data is expected in mid-2026, and topline data from the Embolden trial is anticipated in the second half of 2027. The trial's success depends on 8 of 24 participants meeting Neurogene's composite endpoint.

Stock Titan Editorial Team1mo ago8 min readen

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Key quotes

Exceeded target by dosing a total of 25 participants with NGN-401 in registrational trial due to strong demand from Rett syndrome community

NGN-401 has been generally well-tolerated, with no cases of HLH at the 1E15 vg dose

Topline data from Embolden anticipated in 2H 2027

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