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NGNE

Neurogene Inc.

NGNE

Neurogene Inc. NASDAQ
$21.08 0.43% (+0.09)

Market Cap $326.52 M
52w High $37.27
52w Low $6.88
Dividend Yield 0%
P/E -5.14
Volume 217.40K
Outstanding Shares 15.49M

Income Statement

Period Revenue Operating Expense Net Income Net Profit Margin Earnings Per Share EBITDA
Q3-2025 $0 $23.798M $-20.953M 0% $-0.99 $-20.176M
Q2-2025 $0 $26.081M $-22.016M 0% $-1.05 $-21.248M
Q1-2025 $0 $25.919M $-22.647M 0% $-1.08 $-21.883M
Q4-2024 $0 $6.165M $-19.514M 0% $-1.31 $-20.714M
Q3-2024 $0 $22.18M $-20.217M 0% $-1.19 $-19.407M

Balance Statement

Period Cash & Short-term Total Assets Total Liabilities Total Equity
Q3-2025 $265.41M $287.832M $22.292M $265.54M
Q2-2025 $274.519M $297.316M $24.061M $273.255M
Q1-2025 $292.602M $315.301M $23.506M $291.795M
Q4-2024 $312.405M $335.73M $25.355M $310.375M
Q3-2024 $138.993M $164.12M $26.706M $137.414M

Cash Flow Statement

Period Net Income Cash From Operations Cash From Investing Cash From Financing Net Change Free Cash Flow
Q3-2025 $-20.953M $-19.683M $22.439M $9.858M $12.192M $-19.809M
Q2-2025 $-22.016M $-19.462M $7.45M $54K $-11.958M $-19.61M
Q1-2025 $-22.647M $-20.774M $-45.045M $4K $-65.815M $-21.554M
Q4-2024 $-19.514M $-17.496M $-102.616M $190.065M $69.953M $-17.612M
Q3-2024 $-20.217M $-15.564M $-28.966M $131K $-44.399M $-15.731M

Five-Year Company Overview

Income Statement

Income Statement Neurogene is a classic early‑stage biotech: it has essentially no commercial revenue yet, and its income statement is driven almost entirely by research and development and overhead costs. The company has reported steady, recurring losses over the past several years, which is typical for a clinical‑stage gene therapy business. The size of the loss per share has jumped mainly because of reverse stock splits, not because the business suddenly changed direction. Overall, spending has generally crept higher in line with advancing programs, but not at an uncontrolled pace.


Balance Sheet

Balance Sheet The balance sheet is small in absolute size, reflecting an early‑stage, single‑program‑focused biotech. Assets are dominated by cash and investments, with only modest amounts tied up elsewhere. Debt levels are very low, which limits financial risk from leverage. Shareholders’ equity was negative a couple of years ago but has turned positive again, likely due to capital raising and corporate restructuring, giving the company a better cushion. That said, the capital base is still limited, so financial flexibility depends heavily on access to future funding rather than on existing assets alone.


Cash Flow

Cash Flow Cash flows tell the story of a development‑stage company: consistent cash outflows from operations as the business funds clinical trials, research, and the cost of running its manufacturing capabilities. Free cash flow has been negative each year and closely tracks operating cash burn, since spending on facilities and equipment has been modest. Management has indicated that current cash resources should support operations for a few more years, but over time the company will almost certainly need additional capital unless it secures partners or generates product revenue. The key watchpoint is how quickly cash burn changes as trials scale up.


Competitive Edge

Competitive Edge Neurogene’s competitive position rests on a focused strategy rather than on breadth. Its proprietary EXACT gene regulation platform aims to solve a very specific problem in gene therapy: how to keep protein expression in a safe and effective range, which is particularly important in diseases like Rett syndrome. This gives the company a clear technical angle versus more conventional gene therapies. In‑house manufacturing is another strength, providing more control over quality and timelines in a field where external capacity is often a bottleneck. On the other hand, Neurogene is small, heavily concentrated in a single lead program, and operates in a field where larger players and other innovators are also targeting similar neurological indications. Its moat is therefore promising but unproven, and highly dependent on clinical and regulatory outcomes for its flagship program.


Innovation and R&D

Innovation and R&D Innovation is the core of Neurogene’s story. The EXACT platform is designed to “tune” gene expression, addressing key safety concerns that have held back some central nervous system gene therapies. The lead candidate, NGN‑401 for Rett syndrome, is the first real‑world test of this concept and has shown encouraging early functional gains in patients at the registrational dose, with a safety profile so far consistent with moving forward. At the same time, a serious safety event at a higher dose and the decision to halt advancement of the CLN5 Batten disease program highlight how fragile and unpredictable gene therapy development can be. The pipeline beyond NGN‑401 is still relatively thin, with another program expected to enter the clinic, so R&D is currently concentrated rather than diversified. Future value from innovation will depend on proving long‑term safety and benefit in Rett, successfully broadening the platform to new diseases, and maintaining strong dialogue with regulators.


Summary

Overall, Neurogene is an early‑stage, pre‑revenue gene therapy company with a tightly focused strategy. Financially, it runs steady operating losses funded by a modest but meaningful cash position and minimal debt, implying reliance on capital markets or partnerships over time. Strategically, its strength lies in a differentiated gene regulation platform, in‑house manufacturing, and a clear focus on high‑need neurological disorders, but its fortunes are closely tied to a single lead program, NGN‑401. Recent clinical signals at the registrational dose are encouraging, while setbacks in a higher dose cohort and another program underscore the inherent risks of the space. The key uncertainties are clinical outcomes, regulatory decisions, and future financing, all of which will shape whether the company can turn its promising technology into a sustainable business.