Neurogene Inc. announced on June 30, 2025, that it received written agreement from the U.S. Food and Drug Administration (FDA) on the key elements of its Embolden™ registrational trial design for NGN-401 gene therapy for Rett syndrome. This pivotal single-arm, open-label, baseline-controlled study will evaluate the 1E15 vg dose in females aged three years and older.
The registrational trial's primary endpoint will be a composite of improvement in the Clinical Global Impression-Improvement (CGI-I) scale and the gain of developmental milestones or skills, captured by video. This agreement marks a significant de-risking step for NGN-401, allowing the company to advance towards potential market entry.
Neurogene also confirmed the completion of dosing for the Phase 1/2 clinical trial of NGN-401, with a total of 10 participants having received the 1E15 vg dose, showing no evidence of hemophagocytic lymphohistiocytosis (HLH) or hyperinflammatory syndrome at this dose level. Furthermore, the company announced an extension of its cash runway into early 2028, with $292.6 million in cash, cash equivalents, and investments as of March 31, 2025, following a reallocation of capital to support pivotal trial activities.
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