Neurogene Announces Embolden Trial Design for Rett Syndrome

Neurogene ( (NGNE) ) just unveiled an announcement.

On June 30, 2025, Neurogene announced the design of the Embolden™ registrational trial for NGN-401 gene therapy targeting Rett syndrome, following a written agreement with the FDA. The trial, which will evaluate females aged 3 and older, aims to assess improvements in clinical symptoms and developmental milestones, with the company highlighting the significant potential of NGN-401 to address the needs of the Rett syndrome community. Neurogene’s Phase 1/2 trial data supports the trial design, showing meaningful improvements in participants, and the company remains financially positioned to support operations into early 2028.

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 for rare neurological diseases. The company employs novel approaches to gene therapy, including its proprietary EXACT™ transgene regulation platform, and operates a state-of-the-art gene therapy manufacturing facility in Houston, Texas.

Average Trading Volume: 265,923

Technical Sentiment Signal: Sell

Current Market Cap: $230.5M

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