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Neurogene Begins Dosing in Rett Syndrome Trial

Story Highlights
  • Neurogene dosed the first participant in the Embolden trial for NGN-401 on November 6, 2025.
  • The trial aims to support a future BLA submission and has expanded to 12 of 13 sites.
  • Looking for the best stocks to buy? Follow the recommendations of top-performing analysts.
Neurogene Begins Dosing in Rett Syndrome Trial

Meet Your ETF AI Analyst

Neurogene ( (NGNE) ) just unveiled an update.

On November 6, 2025, Neurogene Inc. announced the dosing of the first participant in the Embolden™ registrational trial of NGN-401, a gene therapy for Rett syndrome. This marks a significant milestone in their efforts to address unmet medical needs in neurodevelopmental disorders. The trial, which is expected to complete enrollment within three to six months, involves 20 females aged three and above, and aims to support a future BLA submission. The company has expanded its U.S. site footprint to meet patient demand, with 12 of 13 clinical sites initiated. NGN-401 has received multiple designations from the FDA and EMA, highlighting its potential impact on the treatment landscape for Rett syndrome.

The most recent analyst rating on (NGNE) stock is a Buy with a $65.00 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 for rare neurological diseases. The company aims to improve the lives of patients and families affected by these conditions through novel gene therapy approaches, including their proprietary EXACT™ transgene regulation platform technology.

Average Trading Volume: 195,405

Technical Sentiment Signal: Hold

Current Market Cap: $453.8M

For an in-depth examination of NGNE stock, go to TipRanks’ Overview page.

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