- Neurogene Inc. announced it will begin **dosing participants in its embolden registrational study this quarter** following completed discussions with the FDA.  **[0001404644_2181093_0]** 
- The company presented results from a **head-to-head non-human primate study** demonstrating that **ICV administration is superior to IT lumbar** for delivering gene therapy to the brain in CNS-mediated disorders, achieving approximately **10 to 100-fold higher drug levels** in key brain areas for Rett syndrome.  **[0001404644_2181093_0]** 
- Neurogene has **expanded its U.S. clinical trial footprint to 13 sites**, more than doubling its previous presence, to meet enrollment demand and facilitate future commercialization.  **[0001404644_2181093_0]** 
- The company emphasized its strategy to prioritize being **"best in class" over "first to market"** for Rett gene therapy, focusing on durable and meaningful gains of function, and highlighted its ability to enroll patients as young as **three years old**, which is younger than competitors.  **[0001404644_2181093_1]** **[0001404644_2181093_4]** **[0001404644_2181093_5]** 
- Neurogene addressed the **valuation gap** with competitors, attributing it to a past safety event and misperceptions regarding ICV delivery and regulatory timelines, asserting that the time to FDA approval for both products is more similar than different, with a potential lead for competitors being at most a quarter.  **[0001404644_2181093_5]**

Neurogene Inc. Provides Updates on Registrational Study, Clinical Data, and Market Strategy

- **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)