olivLaw · US equity research
Neurogene Inc. NGNE
Pharmaceutical Preparations · SIC 2834 · Vertical: Pharmaceutical Preparations (SIC 2834)
Neurogene is a clinical-stage biotechnology company developing genetic therapies for rare neurological diseases, with NGN-401 for Rett syndrome as its lead program. In 2026 the company is preparing a possible marketing application, with its PPQ campaign started and top-line Embolden data expected in the second half of 2027. It has no approved products and no product revenue.
At the close of 05 Nov 2026, Neurogene Inc. (NGNE) stock was 25.65 USD (−7.00% on the day), with market capitalisation 513.3 m USD, beta 1.87, 52-week range 16.37–42.84 USD. Latest reported quarter (Q2 2026): net income of -34.5 m USD, diluted EPS -1.53 USD.
Data as of · AI analysis dated · Source: SEC filings and closing prices, computed by olivLaw.
Price history
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Technical reading
- RSI (14)
- 45.7
- neutral
- Bollinger (20, 2)
- 21.08 – 29.83
- in the upper half of the band · %B 0.52 · band width 34.4%
- SMA 50 / 200
- 29.71 / 27.99
- mixed trend
- MACD (12, 26, 9)
- -0.46 / -0.91
- MACD above signal
Indicators describe past prices; on their own they do not predict direction. On our history the RSI ≤ 40 filter added no return (see the signals methodology).
Performance
| Company | S&P 500 | vs S&P 500 | Vertical median | |
|---|---|---|---|---|
| 1 month | +0.8% | −5.9% | +6.7 pp | — |
| 3 months | −27.9% | −4.6% | −23.3 pp | — |
| 6 months | −17.9% | +0.2% | −18.2 pp | — |
| Year to date | +24.5% | +7.4% | +17.1 pp | — |
| 1 year | −7.5% | +8.2% | −15.7 pp | −11.5% |
| 3 years | +87.5% | +68.4% | +19.1 pp | +38.9% |
| 5 years | −83.3% | +56.5% | −139.9 pp | — |
Price returns, dividends excluded.
Quarterly results
Latest quarter: Q2 2026 · 30 Jun 2026
Quarterly margins
Quarterly statements · USD
| Fiscal year | Q3 '24 | Q4 '24 | Q1 '25 | Q2 '25 | Q3 '25 | Q4 '25 | Q1 '26 | Q2 '26 |
|---|---|---|---|---|---|---|---|---|
| Revenue | 0 | 0 | — | 0 | 0 | 0 | — | — |
| Research & development | 16.3 m | 15.3 m | 17.8 m | 19.4 m | 17.2 m | 20.6 m | 25.2 m | 25.5 m |
| Operating income | -22.2 m | -21.5 m | -25.9 m | -26.1 m | -23.8 m | -27.5 m | -33.3 m | -36.7 m |
| Depreciation & amortisation | 800.0 k | 785.0 k | 749.0 k | 800.0 k | 800.0 k | 776.0 k | 847.0 k | 813.0 k ≈ |
| Interest expense | 3.0 k | 2.0 k | 2.0 k | 1.0 k | 1.0 k | 1.0 k | 0 | 0 |
| Net income | -20.2 m | -19.5 m | -22.6 m | -22.0 m | -21.0 m | -24.7 m | -30.9 m | -34.5 m |
| Diluted EPS | -1.19 | -1.00 ≈ | -1.08 | -1.05 | -0.99 | -1.12 ≈ | -1.39 | -1.53 |
| Operating cash flow | -15.6 m ≈ | -17.5 m | -20.8 m | -19.5 m ≈ | -19.5 m ≈ | -17.4 m | -26.1 m | -23.8 m ≈ |
| Capex | 167.0 k ≈ | 116.0 k | 780.0 k | 148.0 k ≈ | 127.0 k ≈ | 128.0 k | 281.0 k | 354.0 k ≈ |
≈ derived quarter (cumulative difference / share-days average)
E/P over time
E/P = trailing-12-month diluted EPS / price at quarter end. E/P is the inverse of P/E and stays defined when earnings are negative.
Fiscal years
| Fiscal year | 2018 | 2019 | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 |
|---|---|---|---|---|---|---|---|---|
| Revenue | 25.0 m | unavailable | unavailable | unavailable | unavailable | 0 | 925.0 k | 0 |
| Revenue YoY | unavailable | unavailable | unavailable | unavailable | unavailable | unavailable | unavailable | −100.0% |
| Net income | -31.6 m | -69.4 m | -33.3 m | -60.7 m | -55.2 m | -36.3 m | -75.1 m | -90.4 m |
| Net income YoY | unavailable | unavailable | unavailable | unavailable | unavailable | unavailable | unavailable | unavailable |
| Diluted EPS | unavailable | unavailable | -0.64 | -1.10 | -1.04 | unavailable | -4.28 | -4.24 |
| Diluted EPS YoY | unavailable | unavailable | unavailable | unavailable | unavailable | unavailable | unavailable | unavailable |
| net % | -126.3% | unavailable | unavailable | unavailable | unavailable | unavailable | -8,123.7% | unavailable |
| Free cash flow | -31.6 m | -16.3 m | -26.8 m | -50.8 m | -55.1 m | -51.7 m | -71.4 m | -78.4 m |
Reported results vs expectations
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Balance sheet & financial health
Fiscal year 2025 · 31 Dec 2025
- Cash
- 103.8 m USD
- Financial debt
- unavailable USD
- Net debt
- unavailable USD
- Equity
- 264.9 m USD
- Total assets
- 288.6 m USD
- Debt / equity
- unavailable
- Current ratio
- 16.56x
- ROE
- -31.4%
- ROA
- -28.9%
- FCF margin
- unavailable
Financial statements (source on every figure)
Revenue and net income (USD)
Income statement · USD
| Line | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 | Source (latest year) |
|---|---|---|---|---|---|---|---|
| Revenue | — | — | — | 0 | 925.0 k | 0 | SEC (XBRL)· 24 Mar 2026verified |
| Research & development | — | 39.2 m | 47.5 m | 44.4 m | 60.9 m | 75.0 m | SEC (XBRL)· 24 Mar 2026verified |
| Operating income | -33.7 m | -60.7 m | -56.5 m | -55.6 m | -82.6 m | -103.3 m | SEC (XBRL)· 24 Mar 2026verified |
| Depreciation & amortisation | 785.0 k | 1.3 m | 3.2 m | 3.3 m | 3.2 m | 3.0 m | SEC (XBRL)· 24 Mar 2026verified |
| EBITDA | -32.9 m ≈ | -59.4 m ≈ | -53.3 m ≈ | -52.3 m ≈ | -79.4 m ≈ | -100.3 m ≈ | SEC (XBRL)· 24 Mar 2026derived |
| Interest expense | — | — | 2.0 k | 12.0 k | 12.0 k | 5.0 k | SEC (XBRL)· 24 Mar 2026verified |
| Pre-tax income | -33.3 m | -60.7 m | -55.2 m | -36.3 m | -75.1 m | -90.4 m | SEC (XBRL)· 24 Mar 2026verified |
| Income tax | — | — | — | — | 0 | 0 | SEC (XBRL)· 24 Mar 2026verified |
| Net income | -33.3 m | -60.7 m | -55.2 m | -36.3 m | -75.1 m | -90.4 m | SEC (XBRL)· 24 Mar 2026verified |
| Diluted EPS | -0.64 | -1.10 | -1.04 | — | -4.28 | -4.24 | SEC (XBRL)· 24 Mar 2026verified |
| Weighted average diluted shares | 51.8 m | 55.0 m | 55.2 m | — | 17.6 m | 21.3 m | SEC (XBRL)· 24 Mar 2026verified |
≈ computed by olivLaw from the reported lines of the same year: Operating income + Depreciation & amortisation.
Balance sheet · USD
| Line | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 | Source (latest year) |
|---|---|---|---|---|---|---|---|
| Cash | 192.6 m | 142.5 m | 82.0 m | 148.2 m | 136.6 m | 103.8 m | SEC (XBRL)· 24 Mar 2026verified |
| Current assets | 194.5 m | 144.0 m | 84.7 m | 200.3 m | 315.9 m | 271.8 m | SEC (XBRL)· 24 Mar 2026verified |
| Property, plant & equipment, net | 3.6 m | 6.5 m | 20.1 m | 17.2 m | 15.4 m | 12.8 m | SEC (XBRL)· 24 Mar 2026verified |
| Intangibles (excl. goodwill) | 347.0 k | 128.0 k | 0 | — | — | — | SEC (XBRL)· 20 Mar 2023verified |
| Total assets | 210.5 m | 163.3 m | 109.3 m | 222.6 m | 335.7 m | 288.6 m | SEC (XBRL)· 24 Mar 2026verified |
| Accounts payable | 7.2 m | 7.4 m | 625.0 k | 2.6 m | 1.3 m | 2.0 m | SEC (XBRL)· 24 Mar 2026verified |
| Current liabilities | 7.9 m | 8.6 m | 6.7 m | 23.0 m | 15.2 m | 16.4 m | SEC (XBRL)· 24 Mar 2026verified |
| Total liabilities | 19.3 m | 20.4 m | 10.6 m | 36.5 m | 25.4 m | 23.7 m | SEC (XBRL)· 24 Mar 2026verified |
| Equity | 191.2 m † | -91.9 m † | -145.7 m † | 186.0 m † | 310.4 m † | 264.9 m † | SEC (XBRL)· 24 Mar 2026verified |
† equity attributable to the parent's shareholders: in these years the issuer no longer reports the total including non-controlling interests.
Cash flow · USD
| Line | 2020 | 2021 | 2022 | 2023 | 2024 | 2025 | Source (latest year) |
|---|---|---|---|---|---|---|---|
| Operating cash flow | -24.6 m | -47.6 m | -52.8 m | -51.4 m | -70.6 m | -77.2 m | SEC (XBRL)· 24 Mar 2026verified |
| Capex | 2.2 m | 3.3 m | 2.2 m | 321.0 k | 808.0 k | 1.2 m | SEC (XBRL)· 24 Mar 2026verified |
| Free cash flow | -26.8 m ≈ | -50.8 m ≈ | -55.1 m ≈ | -51.7 m ≈ | -71.4 m ≈ | -78.4 m ≈ | SEC (XBRL)· 24 Mar 2026derived |
| Investing cash flow | -2.2 m | -3.3 m | -2.2 m | 25.6 m | -125.3 m | 14.0 m | SEC (XBRL)· 24 Mar 2026verified |
| Financing cash flow | 77.1 m | 732.0 k | 66.5 m | 92.5 m | 184.1 m | 30.4 m | SEC (XBRL)· 24 Mar 2026verified |
≈ computed by olivLaw from the reported lines of the same year: Operating cash flow − Capex.
Computed indicators
Growth
Profitability
Returns
Balance sheet
Liquidity
Cash
Efficiency
Market and valuation
1 indicators that do not apply to the sector.
Valuation
olivLaw classification
- indicators available: 3 of at least 6
- score weight coverage: 18.8% of at least 60%
- valuation methods computed: 0 of at least 1
A scoring methodology on indicators and valuation, not investment advice.
Closing price
05 Nov 2026Source: olivLaw, closing series
Market cap: 513.3 m USD
Key indicators
Coverage and freshness
Score by group
19% · Insufficient data
Estimated value range
unavailable — no valuation method could be computed
Valuation assumptions are editorial choices, not measured; beta is not measured.
Valuation methods
- DCF (discounted cash flow)unavailable · net debt missing: EV cannot be bridged to equity (missing debt is not zero)
- Peers: EV/EBITDAunavailable · net debt missing: EV cannot be bridged to equity (missing debt is not zero)
- Peers: P/Eunavailable · the base of the ratio is not positive
- Dividend discount (DDM)unavailable · the company pays no dividend: a dividend model needs an explicit initiation assumption
no valuation method could be computed
Classification (SIC) comparables group by industry code, not by economic activity; the direct group is an editorial selection by activity. Members' multiples are on their own trailing twelve months, at their own latest close.
Assumptions (illustrative, editorial)
- Risk-free rate
- 4.3%
- Equity risk premium
- 5.0%
- Beta (default)
- 1.00
- Cost of equity
- 9.3%
- Terminal growth
- 2.5%
Factor scorecard
Insufficient data
Minimum-evidence gate: indicators available: 3 of at least 6; score weight coverage: 18.8% of at least 60%; valuation methods computed: 0 of at least 1
| Factor | Value | Score | Weight used |
|---|---|---|---|
| ROIC · Quality | unavailable | unavailable | — |
| Net margin · Quality | unavailable | unavailable | — |
| FCF conversion · Quality | unavailable | unavailable | — |
| Revenue CAGR 3Y · Growth | unavailable | unavailable | — |
| EPS CAGR 3Y · Growth | unavailable | unavailable | — |
| Net debt / EBITDA · Balance sheet | unavailable | unavailable | — |
| Interest coverage · Balance sheet | -60,673.0x | 0 (on a fixed band) | 33% |
| Current ratio · Balance sheet | 10.6x | 100 (on a fixed band) | 22% |
| P/E (TTM) · Valuation | unavailable | unavailable | — |
| EV/EBITDA · Valuation | unavailable | unavailable | — |
| FCF yield · Valuation | -17.1% | 0 (on a fixed band) | 44% |
| Shareholder yield (distributions) · Returns | unavailable | unavailable | — |
| Payout ratio (FCF) · Returns | unavailable | unavailable | — |
Factors without a value were dropped; the remaining weights were renormalised.
Score groups
- Balance sheet40/100 · 2/3 factors with a value
- Growth· partialunavailable · 0/2 factors with a value
- Quality· partialunavailable · 0/3 factors with a value
- Returns· partialunavailable · 0/2 factors with a value
- Valuation· partial0/100 · 1/3 factors with a value
A «partial» group is computed from under half of its weight or from one factor: it is not a full assessment of the group.
Memo
Neurogene Inc. (NGNE). Latest full fiscal year: 2025; data up to 2026-06-30.
Figures computed from the financial statements: revenue 0 USD in 2025; YoY growth -100.0%; ROE -45.3%.
Valuation cannot be computed from the available data.
Insufficient data for a verdict (indicators available: 3 of at least 6; score weight coverage: 18.8% of at least 60%; valuation methods computed: 0 of at least 1).
Text generated deterministically from the figures shown; no language model. Not investment advice.
What supports the thesis
- Current ratio: 10.6x
What contradicts it
- Interest coverage: -60,673.0x
- FCF yield: -17.1%
Not assessed (insufficient data or a ratio without meaning): ROIC, Net margin, FCF conversion, Revenue CAGR 3Y, EPS CAGR 3Y, Net debt / EBITDA, P/E (TTM), EV/EBITDA, Shareholder yield (distributions), Payout ratio (FCF).
Versus the vertical median
Pharmaceutical Preparations (SIC 2834) · same industry (4-digit SIC) · compared with 39 companies
Multiples and returns vs the median
Price rebased to 100 (monthly, 5 years)
Companies in the vertical
| Market cap | P/E | ROE | Net margin | Revenue YoY | 1A / 3A | |
|---|---|---|---|---|---|---|
| LLYEli Lilly And Company | 1,283.9 bn | 44.3 | 86.1% | 31.9% | +44.7% | +27% / +131% |
| JNJJohnson & Johnson | 655.6 bn | 41.3 | 20.0% | 16.2% | +6.0% | +32% / +76% |
| ONCBeone Medicines Ltd. | 575.0 bn | 640.9 | 18.4% | 13.9% | +40.2% | +14% / +107% |
| ABBVAbbVie Inc. | 509.5 bn | 49.6 | — | 15.7% | +8.6% | +27% / +102% |
| MRKMerck & Co., Inc. | 362.2 bn | 151.2 | 5.1% | 3.6% | +1.3% | +40% / +43% |
| ABTAbbott Laboratories | 196.7 bn | 42.3 | 9.2% | 9.7% | +5.7% | −12% / +9% |
| PFEPfizer Inc | 170.3 bn | 42.6 | 4.4% | 6.3% | −1.6% | +16% / −2% |
| VRTXVertex Pharmaceuticals Inc | 137.5 bn | 34.9 | 20.8% | 30.7% | +8.9% | +25% / +53% |
| BMYBristol-Myers Squibb Company | 129.0 bn | 14.0 | 44.1% | 18.5% | −0.2% | +28% / +28% |
| TEVATeva Pharmaceutical Industries Limited | 48.8 bn | 97.3 | 6.6% | 3.6% | +4.3% | +55% / +325% |
| ZTSZoetis Inc. | 33.4 bn | 13.8 | 57.6% | 26.4% | +2.3% | −36% / −54% |
| ROIVRoivant Sciences Ltd. | 26.5 bn | — | -6.2% | -3,535.0% | −71.6% | +76% / +283% |
| RPRXRoyalty Pharma plc | 26.2 bn | 47.7 | 6.9% | 25.7% | +5.1% | +69% / +150% |
| UTHRUnited Therapeutics Corporation | 24.1 bn | 21.3 | 19.1% | 38.6% | +10.6% | +16% / +136% |
| INSMInsmed Inc | 23.9 bn | — | -72.5% | -39.9% | +66.7% | −48% / +335% |
| VTRSViatris Inc. | 21.7 bn | — | -24.3% | -23.9% | −3.0% | +77% / +107% |
| ALNYAlnylam Pharmaceuticals, Inc. | 21.0 bn | 7.9 | 126.6% | 52.8% | +65.2% | −65% / −7% |
| IDYAIDEAYA Biosciences, Inc. | 3.0 bn | — | -38.5% | -1,149.6% | +3,024.4% | −14% / −3% |
| RCUSArcus Biosciences, Inc. | 2.5 bn | — | -114.1% | -453.0% | −4.3% | −32% / +17% |
| STOKStoke Therapeutics, Inc. | 1.7 bn | — | -73.6% | -862.1% | +404.5% | −16% / +588% |
| INVAInnoviva, Inc. | 1.4 bn | 9.7 | 17.0% | 44.1% | +14.7% | −11% / +39% |
| JANXJanux Therapeutics, Inc. | 982.6 m | — | -10.6% | -428.7% | −5.6% | −53% / +84% |
| WVEWave Life Sciences, Inc. | 635.1 m | — | -80.0% | -296.9% | −60.5% | −59% / −39% |
| NGNENeurogene Inc. | 513.3 m | — | -45.3% | — | −100.0% | −7% / +88% |
| DMACDiaMedica Therapeutics Inc. | 487.5 m | — | -114.6% | — | unavailable | +5% / +270% |
1- and 3-year price returns: for NGNE from daily closes (the same as the performance table), for the other companies from month-end closes.
olivLaw engine forecast
The world engine ran after the company's latest material disclosure. Probabilities are of TOUCHING a level within the horizon (not closing there) and sit next to the random walk (p_rw), the no-information benchmark.
The engine has no published run for this company yet.
Valuation scenarios (editable assumptions)
Illustrative scenarios
Scenarios are not available. input data missing
AI analysis
AI-generatedAI-generated analysis (Claude) from cited public sources; it may contain errors. Not investment advice. As of 27 Oct 2026 · Model: claude-haiku-5-5.
In brief
Neurogene is a clinical-stage biotechnology company developing genetic therapies for rare neurological diseases, with NGN-401 for Rett syndrome as its lead program. In 2026 the company is preparing a possible marketing application, with its PPQ campaign started and top-line Embolden data expected in the second half of 2027. It has no approved products and no product revenue.
SWOT
Strengths
- The company has an advanced clinical program with a registrational study under way and significant regulatory designations. Its in-house cGMP facility reduces dependence on third parties for product quality and timing.
- The EXACT platform offers a proprietary mechanism for controlling transgene expression that could extend beyond simple monogenic diseases. Long-term Phase 1/2 data show sustained improvements in all treated participants.
- The company has raised additional capital through public offerings and extended its cash runway into 2029, giving time to prepare a filing.
Weaknesses
- The company has no approved products and no product revenue, and net losses keep rising as research and development spending grows.
- Development depends almost entirely on NGN-401, and alternative programs are at an early stage without a selected candidate.
- The company has not yet completed a pivotal study and, as it acknowledges, has limited experience running such trials.
Opportunities
- If Embolden data confirm the benefit, the company could obtain approval with a broad label and expand patient access.
- The EXACT platform could be applied to other rare neurological diseases, particularly if discovery programs reach clinical candidates.
- Regulatory interest and special designations could shorten review time if the FDA finds the data sufficiently compelling.
Threats
- A failure in the Embolden study could seriously affect the program's prospects and the company's ability to raise funding.
- Safety reactions linked to AAV, including hyperinflammatory syndromes, could cause delays or regulatory restrictions.
- If development and commercialisation costs exceed expectations, the company could be forced to reduce its activities or sell assets, including intellectual property.
Risks
- Single-program dependencehigh
The company's success depends heavily on NGN-401, and a negative result in the Embolden study could affect all other activities.
- Need for additional capitalhigh
The company will need further funding before generating revenue, and the terms of capital access could become less favourable.
- Safety of AAV therapiesmedium
Serious inflammatory reactions, including the fatal case in 2024, could recur in studies or after approval and could change the regulatory timeline.
- Manufacturing and PPQ timingmedium
Completing the PPQ runs by the end of 2026 depends on operational execution, and delays could push back the planned marketing application.
- Reliance on the academic collaboratorlow
Early research depends on the collaboration with the University of Edinburgh, and any breakdown in that relationship could affect the pipeline.
Investment thesis
What would need to be true (bull case)
- If the Embolden study confirms the benefits seen in long-term data, the company could be first to offer a therapy that modifies the course of Rett syndrome, a disease with a clear medical need.
- Regulatory designations and the in-house manufacturing facility could shorten the time to a possible marketing application and give an edge at launch.
- The EXACT platform could generate further programs for rare neurological diseases if they reach the clinic with favourable data.
What would break the thesis
- The company has no revenue and losses keep growing; if Embolden results are delayed or do not confirm the benefit, funding could become more costly.
- Dependence on a single clinical program means a failure could affect the whole company, with no other products to offset it.
- Safety risks linked to AAV therapies, including a death in the earlier study, could lead regulators to ask for more information or restrict use.
What to watch
- Completion of PPQ runs for NGN-401end of 2026
- Top-line data from the Embolden study2027 (second half)
- Submission of the BLA for NGN-4012027
- Development of cash runway and quarterly net lossQ1 2027 (quarterly report)
Products, market & competition
AI-generatedAI-generated analysis (Claude) from cited public sources; it may contain errors. Not investment advice. As of 27 Oct 2026 · Model: claude-haiku-5-5.
The business
Neurogene Inc. is a clinical-stage biotechnology company founded in 2018 that aims to turn devastating neurological diseases into treatable conditions through genetic therapies. The company reports aggregate net proceeds of approximately $552.1 million raised since inception and a net loss of $90.4 million in fiscal 2025 1. It has no products approved for sale and has not generated product revenue 1. Its proprietary EXACT technology uses microRNA-based circuits to limit variability in transgene expression and widen the therapeutic window 1.
The company operates an in-house cGMP facility in Houston, Texas, used for both the Phase 1/2 trial and Embolden, which it says gives better control of product quality and development timelines 1. Its research collaboration with the University of Edinburgh supports discovery work and provides an option to in-license candidates from that laboratory 1. Current activity centres on NGN-401, while early discovery programs have not yet selected a clinical candidate 1.
In August 2026 the company reported that its PPQ campaign for NGN-401 had started, with completion of the PPQ runs expected by the end of 2026 3. A public offering of approximately $144 million gross proceeds extended its cash runway into the first quarter of 2029 3. The CLN5 Batten program was not advanced in its prior form, and the company is evaluating options for it 1.
Segments
NGN-401 (Rett syndrome)
NGN-401 is the lead clinical program, an AAV9 gene therapy delivered intracerebroventricularly that carries the full MECP2 gene and uses the EXACT technology 1. The Embolden registrational study evaluates the 1E15 vg dose in 20 females, with a minimum response rate of 33% set as the success threshold in the latest filing 2. As of June 2026, all 10 participants in the Phase 1/2 study at this dose had improved on CGI-I and gained at least one developmental milestone 3.
Market context
Rett syndrome has an estimated global incidence of 1 in 10,000 females, and prevalence in the United States, European Union and United Kingdom is estimated at 15,000 to 20,000 patients 1. One treatment is approved but is not disease modifying, leaving a significant unmet need 1. The sources do not give a revenue-based market size, so the commercial scale remains undetermined from these documents.
The market for genetic therapies for rare neurological diseases depends on clinical results, the timing of approvals and the ability of companies to fund operations over the long term. Companies in this segment often rely on capital raising, and trial outcomes can materially change near-term prospects 1.
Trends
- Accelerated regulatory designationstailwind
NGN-401 has received several designations, including Breakthrough Therapy from the FDA, which could speed review if the data continue to support clinical benefit 3. The RMAT designation and participation in the FDA's START program give more frequent contact with the regulator 1.
- Interest in direct brain deliverytailwind
Intracerebroventricular administration aims for better distribution in affected regions of the brain, which could become a point of differentiation for neurological gene therapies 1.
- Safety risks linked to AAVheadwind
A participant in the higher-dose cohort died in 2024 from a hyperinflammatory syndrome linked to systemic AAV exposure, and the company discontinued that dose 1. Such syndromes remain a general risk for AAV therapies and can influence regulatory decisions 1.
- Reliance on capital marketsmixed
The company has raised capital several times and depends on capital markets to fund losses, which reached $34.5 million in a single quarter 3. Access to funding could become harder if clinical data fall short of expectations.
Competitive position
The sources do not describe direct competitors by name or product. They note that one treatment is approved for Rett syndrome, but it is not considered disease modifying 1. The company's claimed advantage rests on direct brain delivery and control of transgene expression 1.
Genetic therapies for other neurological diseases could compete for the same investor attention and research resources, though the sources do not name them. The company says it differentiates through the EXACT platform and a biology-first design approach 1.
- Approved treatment for Rett syndrome
One approved treatment exists but does not modify the course of the disease, which leaves room for therapies that target the genetic cause 1.
- Other AAV gene therapies for neurological diseases
The company mentions other AAV therapies that encountered systemic inflammation problems, without naming them in the analysed sources 1.
- University of Edinburgh (academic collaborator)
Not a competitor but a research partner that supports platform development and early candidates 1.
- Other genetic therapy companies in neurology
The sources do not name them, but the company notes that conventional gene therapy is limited to a small number of monogenic diseases 1.
Links, dependencies and risks
Who the company is tied to, from its own SEC filings (customers, suppliers, production, partners, competitors, countries, regulation), from the 5 %+ holders' statements (13D/13G) and from what other companies say about it. Click a node for its evidence; on a listed company the graph moves to it.
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News & filings
In the press
No news in our archive over the last 180 days.
SEC filings
- Quarterly report11 Aug 2026
- Current report11 Aug 2026Results of operations · Financial statements and exhibits
- Current report02 Jul 2026Material agreement · Other events · Financial statements and exhibits
- Current report29 Jun 2026Regulation FD disclosure · Other events · Financial statements and exhibits
- Current report08 Jun 2026Regulation FD disclosure · Financial statements and exhibits
- Current report04 Jun 2026Shareholder vote
- Current report12 May 2026Results of operations · Financial statements and exhibits
- Quarterly report12 May 2026
- Current report20 Apr 2026Departure or appointment of officers · Regulation FD disclosure · Financial statements and exhibits
- Proxy statement16 Apr 2026
Sources
- [1] Form 10-K, fiscal year ended 2025-12-31 · SEC / Neurogene Inc. · 24 Mar 2026
- [2] Form 10-Q, quarter ended 2026-06-30 · SEC / Neurogene Inc. · 11 Aug 2026
- [3] Form 8-K, results release (exhibit 99.1) · SEC / Neurogene Inc. · 11 Aug 2026
Figures computed by olivLaw from SEC filings and closing prices; the qualitative analysis is AI-generated from cited sources. Not investment advice.