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Neurogene Completes FDA Talks for Rett Syndrome Trial

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Neurogene Completes FDA Talks for Rett Syndrome Trial

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An announcement from Neurogene ( (NGNE) ) is now available.

On October 9, 2025, Neurogene Inc. announced the completion of discussions with the FDA regarding the protocol for its Embolden™ registrational trial for NGN-401 gene therapy in Rett syndrome, with plans to begin dosing in the fourth quarter of 2025. The company presented new preclinical data at the ESGCT Congress, demonstrating that intracerebroventricular (ICV) administration of NGN-401 is superior to intrathecal lumbar (IT-L) in reaching key brain regions, reinforcing the potential efficacy of this delivery method. The trial will enroll participants across 13 sites, reflecting strong interest from the Rett syndrome community, and aims to expedite the submission of a BLA application for NGN-401.

The most recent analyst rating on (NGNE) stock is a Buy with a $45.00 price target. To see the full list of analyst forecasts on Neurogene stock, see the NGNE Stock Forecast page.

Spark’s Take on NGNE Stock

According to Spark, TipRanks’ AI Analyst, NGNE is a Underperform.

Neurogene’s overall score is primarily influenced by its financial performance, which is challenged by zero revenues and significant net losses. While the stock shows some technical strength, the high beta and potential overbought conditions pose risks. The valuation reflects the speculative nature of investing in an early-stage biotech firm.

To see Spark’s full report on NGNE stock, click here.

More about Neurogene

Neurogene Inc. is a clinical-stage company focused on developing genetic medicines to treat rare neurological diseases. The company is working on novel approaches to address the limitations of conventional gene therapy, particularly for central nervous system disorders, and has developed the proprietary EXACT™ transgene regulation platform technology. Neurogene has a gene therapy manufacturing facility in Houston, Texas, supporting its clinical development activities.

Average Trading Volume: 151,993

Technical Sentiment Signal: Sell

Current Market Cap: $335M

Find detailed analytics on NGNE stock on TipRanks’ Stock Analysis page.

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