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Neurogene outlines 2026 milestones for NGN-401 Rett therapy

Story Highlights
  • Neurogene advanced NGN-401 for Rett syndrome in late 2025, dosing multiple Embolden trial participants and expanding to 13 U.S. sites.
  • For 2026, Neurogene plans to complete Embolden dosing, present interim NGN-401 data, and intensify commercial-readiness as cash runway extends to early 2028.
  • Looking for the best stocks to buy? Follow the recommendations of top-performing analysts.
Neurogene outlines 2026 milestones for NGN-401 Rett therapy

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The latest update is out from Neurogene ( (NGNE) ).

On January 12, 2026, Neurogene reported recent progress and 2026 milestones for NGN-401, its investigational AAV9 gene therapy targeting Rett syndrome, highlighting that multiple participants were dosed in the Embolden registrational trial during the fourth quarter of 2025 and that 13 U.S. trial sites have been initiated. The company noted previously reported positive interim Phase 1/2 data showing durable multidomain improvements across a wide range of disease severity, outlined plans to complete Embolden dosing in the second quarter of 2026 and present mid-2026 interim safety and efficacy updates across pediatric and adolescent/adult cohorts, and said it has begun early commercial-readiness efforts, supported by in-house manufacturing capabilities and cash runway expected through the first quarter of 2028, underscoring NGN-401’s growing clinical momentum and potential to strengthen Neurogene’s position in gene therapies for rare neurological conditions.

The most recent analyst rating on (NGNE) stock is a Hold with a $20.00 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 Neutral.

The score is held back primarily by weak financial performance (no revenue, widening losses, and heavy cash burn), with additional drag from negative technical momentum and limited valuation support due to losses. Offsetting these risks are a strong, low-debt balance sheet and constructive recent clinical/regulatory milestones for NGN-401.

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

More about Neurogene

Neurogene Inc. is a New York-based clinical-stage biotechnology company focused on developing genetic medicines for rare, devastating neurological diseases. The company is advancing novel gene therapy approaches for central nervous system disorders, leveraging its proprietary EXACT transgene regulation platform to optimize potency and safety, and operates a state-of-the-art gene therapy manufacturing facility in Houston that produces CGMP material for pivotal clinical development, including its lead Rett syndrome candidate NGN-401.

Average Trading Volume: 220,139

Technical Sentiment Signal: Strong Sell

Current Market Cap: $289.3M

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

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