- **Neurogene Inc.** has completed discussions with the FDA on the protocol for its Embolden™ registrational trial for **NGN-401 gene therapy** in Rett Syndrome.  **[1404644_0001404644-25-000088_ngne_prxngn-401updateesgct.htm:0]** 
- The company plans to initiate dosing in the registrational trial in **Q4 2025**, with enrollment expected across **13 sites**.  **[1404644_0001404644-25-000088_ngne_prxngn-401updateesgct.htm:0]** **[1404644_0001404644-25-000088_ngne-20251009.htm:1]** 
- New preclinical data demonstrated that intracerebroventricular (ICV) delivery of NGN-401 achieved **superior AAV biodistribution** in key brain regions compared to intrathecal lumbar (IT-L) delivery.  **[1404644_0001404644-25-000088_ngne_prxngn-401updateesgct.htm:0]** **[1404644_0001404644-25-000088_ngne-20251009.htm:1]** 
- The first four participants in the NGN-401 Phase 1/2 trial gained **23 developmental milestones/skills**, representing multi-domain improvement.  **[1404644_0001404644-25-000088_ngne_prxngn-401updateesgct.htm:0]** 
- Neurogene's cash runway is extended to fund operations into **early 2028**.  **[1404644_0001404644-25-000088_a202510_ngnecorporatepre.htm:4]** **[1404644_0001404644-25-000088_a202510_ngnecorporatepre.htm:11]**

Neurogene (NGNE)