Neurogene (NGNE) AI Stock Analysis

• Market Cap: $321.26M
• Dividend Yield: N/A
• Average Volume (3M): 225.72K
• Price to Earnings (P/E): ―
• Beta (1Y): 3.10
• Revenue Growth: -100.00%
• EPS Growth: 1.47%
• Country: US
• Employees: 107
• Sector: Healthcare
• Sector Strength: 45
• Industry: Biotechnology
• EPS (TTM): -0.99
• Shares Outstanding: 15,489,800
• 10 Day Avg. Volume: 272,921
• 30 Day Avg. Volume: 225,725
• PEG Ratio: -0.10
• Price to Book (P/B): 1.29
• Price to Sales (P/S): 434.14
• P/FCF Ratio: -5.62
• Enterprise Value/Market Cap: 0.81
• Enterprise Value/Revenue: N/A
• Enterprise Value/Gross Profit: -17.04
• Enterprise Value/Ebitda: -3.11
• 1Y Price Target: $83.20
• Price Target Upside: 301.16% Upside
• Rating Consensus: Strong Buy
• Number of Analyst Covering: 6
• EPS Forecast (FY): -5.01
• Revenue Forecast (FY): N/A

• Rating: 48 Neutral
• Price Target: $19.00 ▼(-8.39% Downside)
• Action: Reiterated Date: 01/12/26
• Date: Action: Reiterated 01/12/26

The score is held down primarily by weak financial performance (no TTM revenue, widening losses, and sizable cash burn) and bearish technicals (trading below key moving averages with negative MACD). These are partially offset by a strong balance sheet and positive clinical/regulatory progress in NGN-401 that supports the longer-term thesis but does not yet reduce near-term execution and funding risk.

Positive Factors

Low leverage / strong balance sheet

Substantial equity and minimal debt provide durable solvency and financial flexibility. This capital base supports ongoing R&D and clinical programs, reduces immediate refinancing pressure, and gives management runway to execute registrational activities without near-term liquidity distress.

Advancing registrational trial and positive interim data

Progressing a registrational trial with supportive interim data materially de-risks the lead program over time. Successful mid-stage results and active dosing increase the likelihood of a future BLA, strengthen competitive position in rare neurogene therapies, and justify continued development investment.

Cash runway and in-house manufacturing

Having in-house manufacturing plus a multi-year runway is a durable operational advantage: it improves control over supply, reduces commercialization bottlenecks, and gives time to reach pivotal milestones before needing large external funding, lowering near-term execution risk.

Negative Factors

Pre-revenue with widening losses

Continued absence of product revenue combined with growing operating losses erodes equity and limits internal funding options. Over months this increases dependence on external financing or partnerships and delays any path to sustainable profitability if R&D spend remains elevated.

High and accelerating cash burn

Sustained negative operating and free cash flow creates ongoing financing need; even with current runway, accelerating burn heightens dilution or strategic-partner reliance risk. Over a multi-quarter horizon this can constrain R&D flexibility and force value-destructive capital raises.

Concentration risk on single lead program

Business relies heavily on the success and timing of one lead gene-therapy program. This creates a binary outcome exposure: trial delays, adverse results, or regulatory setbacks would materially damage revenue prospects and valuation, making the company's near-to-mid-term trajectory highly outcome-dependent.

Neurogene Business Overview & Revenue Model

Company Description

Neurogene Inc. develops life-changing genetic medicines for patients and their families affected by neurological diseases. Its product candidate includes NGN-401, an investigational AAV9 gene therapy for the treatment of Rett syndrome; and NGN-101 to treat neuronal ceroid lipofuscinosis subtype 5 batten disease. The company is headquartered in New York, New York.

How the Company Makes Money

Neurogene makes money primarily through the development and commercialization of its gene therapy products. The company's revenue streams include licensing agreements, where it partners with larger pharmaceutical companies to co-develop and market its therapies, and potential sales of approved treatments. Neurogene may also receive funding through research grants, collaborations with academic institutions, and milestone payments from its strategic partnerships, which support its ongoing research and development efforts.

Neurogene Financial Statement Overview

Summary

Financials are mixed: a strong, low-debt balance sheet (debt ~$11.3M vs. equity ~$265.5M) supports solvency, but the business is effectively pre-revenue (TTM revenue $0) with widening losses (TTM net loss ~$85.1M) and heavy cash burn (TTM FCF about -$78.6M), increasing longer-term financing risk if burn persists.

Income Statement

NGNE remains a pre-revenue/early-commercial business, with revenue at $0 in TTM (Trailing-Twelve-Months) versus $0.9M in 2024. Profitability is very weak: TTM shows a net loss of ~$85.1M (vs. ~$75.1M in 2024), and operating losses have expanded from 2023 levels. While losses are typical for biotechnology at this stage, the trend points to rising spend without a corresponding revenue ramp, keeping earnings quality and near-term profitability outlook pressured.

Balance Sheet

The balance sheet is a clear strength. Leverage is low, with total debt of ~$11.3M against stockholders’ equity of ~$265.5M in TTM (Trailing-Twelve-Months), implying modest balance-sheet risk. Total assets are ~$287.8M, providing a sizable capital base to fund operations. The key weakness is returns: equity is being consumed by ongoing losses (negative return on equity in TTM), so while solvency looks solid today, continued losses could erode this cushion over time.

Cash Flow

Cash generation is weak, with TTM (Trailing-Twelve-Months) operating cash flow of about -$77.4M and free cash flow of about -$78.6M, reflecting a meaningful cash burn profile. Free cash flow was slightly worse than 2024 (about -$71.4M), indicating accelerating cash usage. A positive offset is that free cash flow broadly tracks net losses (free cash flow roughly in line with net income), suggesting the burn is not being driven by large working-capital swings—but the magnitude of outflows still implies reliance on existing capital and/or future financing.

Breakdown	TTM	Dec 2024	Dec 2023	Dec 2022	Dec 2021	Dec 2020
Income Statement
Total Revenue	0.00	925.00K	0.00	0.00	0.00	0.00
Gross Profit	-15.35M	-2.32M	-3.26M	-3.20M	-42.26M	-1.82M
EBITDA	-84.02M	-71.89M	-52.29M	-51.99M	-49.59M	-31.46M
Net Income	-85.13M	-75.14M	-36.32M	-55.19M	-50.52M	-33.28M
Balance Sheet
Total Assets	287.83M	335.73M	222.57M	109.27M	101.49M	210.52M
Cash, Cash Equivalents and Short-Term Investments	265.41M	312.40M	197.16M	82.02M	70.54M	192.56M
Total Debt	11.27M	12.43M	16.25M	4.69M	6.43M	12.12M
Total Liabilities	22.29M	25.36M	36.55M	10.64M	193.36M	19.30M
Stockholders Equity	265.54M	310.38M	186.02M	98.63M	-91.88M	191.22M
Cash Flow
Free Cash Flow	-78.58M	-71.41M	-51.74M	-55.05M	-64.77M	-26.79M
Operating Cash Flow	-77.41M	-70.60M	-51.42M	-52.82M	-46.40M	-24.57M
Investing Cash Flow	-117.77M	-125.26M	25.64M	-2.23M	-18.37M	-2.22M
Financing Cash Flow	199.98M	184.07M	92.48M	66.53M	51.06M	77.14M

Neurogene Technical Analysis

• Technical Analysis Sentiment: Positive
• Last Price: 20.74
• 50DMA: Positive
• 100DMA: Positive
• 200DMA: Positive
• MACD: Negative
• RSI: Negative
• STOCH: Negative

Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For NGNE, the sentiment is Positive. The current price of 20.74 is above the 20-day moving average (MA) of 18.09, above the 50-day MA of 18.71, and below the 200-day MA of 21.00, indicating a bullish trend. The MACD of 0.52 indicates Negative momentum. The RSI at 71.09 is Negative, neither overbought nor oversold. The STOCH value of 82.24 is Negative, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Positive sentiment for NGNE.

Neurogene Peers Comparison

Name	Overall Rating	Market Cap	P/E Ratio	ROE	Dividend Yield	Revenue Growth	EPS Growth
AVIR	58 Neutral	$380.48M	-2.74	-37.98%	―	―	14.33%
Sector *	51 Neutral	$7.86B	-0.30	-43.30%	2.27%	22.53%	-2.21%
VOR	50 Neutral	$660.17M	-0.04	―	―	―	-1072.48%
NGNE	48 Neutral	$321.26M	-4.68	-42.25%	―	-100.00%	1.47%
SLN	47 Neutral	$282.46M	-3.58	―	―	―	―
ELTX	43 Neutral	$242.05M	-4.07	―	―	―	17.36%
TNYA	43 Neutral	$123.41M	-0.72	-96.44%	―	―	46.68%

Neurogene Corporate Events

Neurogene outlines 2026 milestones for NGN-401 Rett therapy

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.

Neurogene Announces Positive Interim Data for NGN-401

On November 12, 2025, Neurogene Inc. announced positive interim clinical data from its Phase 1/2 trial of NGN-401 gene therapy for females with Rett syndrome, showing significant functional gains in pediatric participants. The results indicate durable improvements across multiple domains, suggesting NGN-401’s potential as a best-in-class gene therapy, with no severe adverse events reported, enhancing Neurogene’s position in the gene therapy market for neurological disorders.

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.

Neurogene Begins Dosing in Rett Syndrome Trial

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.