Neurogene completes dosing in gene therapy trial for Rett syndrome
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Neurogene Announces Successful Completion of Dosing in Embolden™ Registrational Trial of NGN-401 for Rett Syndrome
June 8, 2026 7:30 AM EDTExceeded 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; additional interim Phase 1/2 data expected mid-2026
Topline data from Embolden anticipated in 2H 2027
NEW YORK--(BUSINESS WIRE)-- 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 successful completion... More
