Neurogene Inc. (NASDAQ:NGNE) shares are up during Friday’s premarket session following the announcement that the FDA has granted Breakthrough Therapy designation for NGN-401, an investigational gene therapy for Rett syndrome, a rare neurological disorder caused by genetic mutations that disrupt brain development, primarily affecting girls.
The Breakthrough Therapy designation was awarded based on interim data from the Phase 1/2 trial, which demonstrated clinically meaningful improvements across multiple functional domains in patients with Rett syndrome.
The company is on track to complete dosing in its Embolden registrational trial by the second quarter of 2026 and plans to present additional interim clinical data in mid-2026.
The FDA’s decision underscores the significant unmet medical need for effective treatments for Rett syndrome, as NGN-401 aims to deliver the full-length human MECP2 gene using Neurogene’s proprietary EXACT transgene regulation technology.
The designation allows for expedited development and review processes, including eligibility for Priority Review and rolling submission of the Biologics License Application.
Technical Analysis
Currently, the stock is trading 34.30% above its previous close, reflecting strong momentum following the positive news. Shares have increased significantly over the past 12 months, and they are currently positioned closer to their 52-week highs than lows.
The RSI is at 50.00, which is considered neutral territory, indicating that the stock is neither overbought nor oversold. Meanwhile, MACD is at 0.10, below its signal line at 0.15, suggesting bearish pressure on the stock.
The combination of neutral RSI and bearish MACD indicates mixed momentum for NGNE.
- Key Resistance: $37.50
- Key Support: $25.00
Analyst Consensus & Recent Actions: The stock carries a Strong Buy rating with an average price target of $48.33. Recent analyst moves include:
- LifeSci Capital: Initiated with Outperform (Target $50.00) (Jan. 26)
- HC Wainwright & Co.: Buy (Maintains Target to $70.00) (Jan. 13)
NGNE Price Action: Neurogene shares were up 23.37% at $24.28 during premarket trading on Friday, according to Benzinga Pro data.
Image via Shutterstock
