NGNE on Nasdaq

Neurogene Inc.

Pharmaceutical preparations company, listed on Nasdaq (NGNE)1

In its 2026 annual report the company says: “We are a clinical-stage biotechnology company committed to overcoming these limitations and turning today’s complex devastating neurological diseases into treatable conditions. We are building a robust and differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet need not otherwise addressable by conventional gene therapy.”3 It is headquartered in New York, New York.1

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Last updated Oct 21, 2026.

Overview

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  • TickerNGNE on Nasdaq1
  • IndustryPharmaceutical Preparations1
  • HeadquartersNew York, New York1
  • Business address535 W 24Th Street, 5Th Floor, New York, Ny 100112
  • Filer categoryNon-accelerated filer, Smaller reporting company2

Despite recent scientific advances in genetics, most neurological diseases, particularly those with devastating consequences to patients, are left untreated. Conventional gene therapy is an attractive potential treatment approach for only a limited number of monogenic diseases due to the challenges caused by the complex biology of neurological diseases and by inherent variable transgene uptake and expression. We are a clinical-stage biotechnology company committed to overcoming these limitations and turning today’s complex devastating neurological diseases into treatable conditions. We are building a robust and differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet need not otherwise addressable by conventional gene therapy. One approach we are taking harnesses our proprietary transgene regulation technology, EXACT TM (Expression Attenuation …3 From its latest annual report

Financials

From the company’s reports to the SEC. Every number links to the filing it came from.

Key numbers, fiscal 2025

-100.0%Revenue growth, 20254
-34.1%Return on equity4
-$78.4MFree cash flow4

Financial statements

Revenue54$0 in 2025
0$250.0K$500.0K$750.0K$1.0M2023: $0$020232024: $925.0K$925.0K20242025: $0$02025
Operating income678954-$103.3M in 2025+46% since 2019
-$150.0M-$100.0M-$50.0M02019: -$71.0M-$71.0M20192020: -$33.7M-$33.7M2020−52%2021: -$60.7M-$60.7M2021+80%2022: -$56.5M-$56.5M2022−7%2023: -$55.6M-$55.6M2023−2%2024: -$82.6M-$82.6M2024+49%2025: -$103.3M-$103.3M2025+25%
Net income678954-$90.4M in 2025+30% since 2019
-$100.0M-$75.0M-$50.0M-$25.0M02019: -$69.4M-$69.4M20192020: -$33.3M-$33.3M2020−52%2021: -$60.7M-$60.7M2021+82%2022: -$55.2M-$55.2M2022−9%2023: -$36.3M-$36.3M2023−34%2024: -$75.1M-$75.1M2024+107%2025: -$90.4M-$90.4M2025+20%

Every line of the income statement as filed: the last 2 fiscal years, latest first.4

Period ending2025-12-312024-12-31
Revenue under licensing agreements$0$925.0K
Research and development expenses$75.0M$60.9M
General and administrative expenses$28.3M$22.6M
Total operating expenses$103.3M$83.5M
Loss from operations-$103.3M-$82.6M
Interest income$11.5M$8.5M
Interest expense-$5.0K-$12.0K
Other income$1.7M$574.0K
Other expense-$230.0K-$1.6M
Net loss-$90.4M-$75.1M
Net loss per share, basic (in dollars per share)-$4.24-$4.28
Net loss per share, diluted (in dollars per share)-$4.24-$4.28
Weighted-average shares of common stock outstanding, basic (in shares)21.3M shares17.6M shares
Weighted-average shares of common stock outstanding, diluted (in shares)21.3M shares17.6M shares

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Corporate structure

Neurogene Inc. sits at the top of its group. 1 entity on record. Switch between the flow chart and the network, and select any entity to see its details.

Major holders of Neurogene Inc.

  • Prudential Financial Filed SCHEDULE 13G on Neurogene Inc., latest Oct 7, 2026
  • JENNISON ASSOCIATES LLC Filed SCHEDULE 13G on Neurogene Inc., latest Oct 6, 2026
  • BALYASNY ASSET MANAGEMENT L.P. Filed SCHEDULE 13G on Neurogene Inc., latest Aug 14, 2026
  • Redmile Group, LLC Filed SCHEDULE 13G/A on Neurogene Inc., latest Aug 14, 2026 (8 filings since Feb 14, 2022)
  • Trails Edge Capital Partners, LP Filed SCHEDULE 13G/A on Neurogene Inc., latest Aug 14, 2026 (2 filings since Dec 15, 2025)
  • EcoR1 Capital, LLC Filed SCHEDULE 13G/A on Neurogene Inc., latest Nov 14, 2025 (3 filings since Feb 14, 2022)
  • BAKER BROS. ADVISORS LP Filed SCHEDULE 13G/A on Neurogene Inc., latest Aug 14, 2025 (6 filings since Jul 19, 2023)
  • Commodore Capital LP Filed SCHEDULE 13G/A on Neurogene Inc., latest Aug 14, 2025 (2 filings since Dec 11, 2024)
  • FMR LLC Filed SCHEDULE 13G/A on Neurogene Inc., latest May 12, 2025 (2 filings since Nov 12, 2024)
  • BlackRock Filed SCHEDULE 13G/A on Neurogene Inc., latest Apr 23, 2025 (4 filings since Feb 3, 2022)
  • Casdin Capital, LLC Filed SCHEDULE 13G/A on Neurogene Inc., latest Feb 14, 2025 (2 filings since Nov 8, 2024)
  • GREAT POINT PARTNERS LLC Filed SCHEDULE 13G/A on Neurogene Inc., latest Feb 14, 2025 (4 filings since Dec 27, 2023)
  • Neurogene Inc. New York, New York

Built from major holders from Schedules 13D and 13G. Each entity in the chart links to its source.

What it owns

What Neurogene Inc. reports owning in its annual reports to the SEC: buildings, land and equipment, real estate and locations, investments, intangible assets and inventory, at the end of its latest fiscal year and a year before.

Property and equipment

ItemLatestA year beforeChange
Property and equipment, before depreciation$26.9M4 Dec 31, 2025$26.6M4 Dec 31, 2024+1.2%
Accumulated depreciation$14.1M4 Dec 31, 2025$11.2M4 Dec 31, 2024+26.3%
Property and equipment, net$12.8M4 Dec 31, 2025$15.4M4 Dec 31, 2024−17.1%
Finance-lease assets$22.0K4 Dec 31, 2025$71.0K4 Dec 31, 2024−69.0%
Operating-lease assets (leased space)$2.6M4 Dec 31, 2025$3.0M4 Dec 31, 2024−11.7%

Investments

ItemLatestA year beforeChange
Cash and cash equivalents$103.8M4 Dec 31, 2025$136.6M4 Dec 31, 2024−24.0%
Marketable securities, current$165.2M4 Dec 31, 2025$175.8M4 Dec 31, 2024−6.1%

Properties, in its own words

We currently lease two properties for our principal offices: an approximately 42,000 square foot manufacturing facility in Houston, Texas, and an approximately 6,000 square foot office space in New York, New York. The Houston manufacturing facility is used for our in-house manufacturing, warehouse and cold storage functions, and the lease expires on August 31, 2029. The New York office space is our corporate headquarters. In November 2025, we extended the lease by an additional 19 months at a reduced cost per square foot, which now expires on January 31, 2028. In addition, we have established a hybrid work-from-home policy for many of our employees. We believe these spaces to be sufficient to meet our needs for the foreseeable future and that any additional space we may require will be available on commercially reasonable terms.

In addition to our principal offices, we also lease a combined 39,572 square feet of office and laboratory space in Seattle, Washington that was previously used by Neoleukin prior to the merger for laboratory, discovery, research and development and general and administrative purposes. We have entered into a sublease with an unrelated third party for 6,272 square feet of that space, which will terminate concurrently with the end of our lease for such space on September 30, 2026. We are actively looking for a subtenant for the remaining 33,300 square feet of combined laboratory and office space, which is subject to a lease that will terminate on June 30, 2029. Any net proceeds we receive from any sublease of these properties will be payable to the holders of CVRs issued in connection with the merger, after adjustment for certain costs.

Item 2 of the 10-K filed Mar 24, 2026.3

Leadership

Who has led Neurogene Inc., and when. Pick a name to see everywhere that person has worked.

Board members and other officers (34)

Former names

  • Neoleukin Therapeutics, Inc.Aug 12, 2019 to Dec 6, 20232
  • AQUINOX PHARMACEUTICALS, INCDec 19, 2013 to Aug 12, 20192
  • AQUINOX PHARMACEUTICALS (USA) INCJun 25, 2007 to Dec 24, 20132

Timeline

  1. Aug 11, 20268-K Results of operations49
  2. Jul 2, 20268-K Entered a material agreement; Other events50
  3. Jun 29, 20268-K Regulation FD disclosure; Other events51
  4. Jun 8, 20268-K Regulation FD disclosure52
  5. Jun 4, 20268-K Shareholder vote53
  6. May 12, 20268-K Results of operations54
  7. Apr 20, 20268-K Executive or director change; Regulation FD disclosure55
  8. Mar 24, 20268-K Results of operations56
  9. Feb 26, 20268-K Regulation FD disclosure57
  10. Jan 12, 20268-K Regulation FD disclosure58
  11. Nov 13, 20258-K Results of operations59
  12. Nov 12, 20258-K Regulation FD disclosure60
  13. Dec 26, 2023New chief executive Rachel McMinn becomes CEO12
  14. Mar 6, 2014New chief executive David Main becomes CEO13

Calendar

Coming up

  1. Nov 12, 2026Reports results for the quarter ending Sep/2026 A year earlier it reported ($0.99) a share, on Nov 13, 2025.61

When it reported results

  1. Aug 11, 2026Reported quarterly or annual results49
  2. May 12, 2026Reported quarterly or annual results54
  3. Mar 24, 2026Reported quarterly or annual results56
  4. Nov 13, 2025Reported quarterly or annual results59
  5. Aug 11, 2025Reported quarterly or annual results62
  6. May 9, 2025Reported quarterly or annual results63
  7. Mar 24, 2025Reported quarterly or annual results64
  8. Nov 18, 2024Reported quarterly or annual results65

Upcoming dates from Nasdaq's calendar; past results from the company's 8-K filings (Item 2.02). The full markets calendar

Filings

307 filings to the SEC since October 2021: 5 annual reports, 15 quarterly reports, 66 major events, 97 insider forms, 56 large holders, 68 other. The latest 20:

  1. Oct 7, 2026SCHEDULE 13G EDGAR
  2. Oct 6, 2026SCHEDULE 13G EDGAR
  3. Oct 2, 20264 EDGAR
  4. Aug 14, 2026SCHEDULE 13G/A EDGAR
  5. Aug 14, 2026SCHEDULE 13G EDGAR
  6. Aug 14, 2026SCHEDULE 13G/A EDGAR
  7. Aug 11, 202610-Q EDGAR
  8. Aug 11, 20268-K EDGAR
  9. Jul 7, 20264 EDGAR
  10. Jul 2, 2026424B5 EDGAR
  11. Jul 2, 20264 EDGAR
  12. Jul 2, 20268-K EDGAR
  13. Jun 30, 2026424B5 EDGAR
  14. Jun 29, 20268-K EDGAR
  15. Jun 16, 20264/A EDGAR
  16. Jun 16, 20264/A EDGAR
  17. Jun 16, 20264/A EDGAR
  18. Jun 16, 20263/A EDGAR
  19. Jun 8, 20268-K EDGAR
  20. Jun 4, 20268-K EDGAR

The 307 filings since October 2021 can be filtered by form type and year here when JavaScript is on.

Every filing from the last five years, summarized. The full record is on SEC EDGAR.

Insider trades

25 open-market trades by officers, directors and large owners since October 2021: 6 buys and 19 sales. Grants, option exercises and gifts are left out.

DateWhoTradeSharesPriceValueFiling
Jul 7, 2026Cvijic Christine MikailPresident and CFOSale4,200$36.13$151.7KForm 4
Jul 6, 2026Cvijic Christine MikailPresident and CFOSale600$36.02$21.6KForm 4
Jul 2, 2026Cvijic Christine MikailPresident and CFOSale4,061$34.82$141.4KForm 4
Jul 1, 2026Cvijic Christine MikailPresident and CFOSale739$34.06$25.2KForm 4
May 6, 2026Cvijic Christine MikailPresident and CFOSale9,600$31.25$300.0KForm 4
Mar 26, 2026Jordan JulieChief Medical OfficerSale828$21.89$18.1KForm 4
Mar 26, 2026McMinn RachelChief Executive OfficerSale3,352$22.07$74.0KForm 4
Mar 26, 2026Cvijic Christine MikailPresident and CFOSale2,558$21.62$55.3KForm 4
Mar 13, 2026Cobb StuartChief Scientific OfficerSale6,797$20.81$141.4KForm 4
Mar 13, 2026Cvijic Christine MikailPresident and CFOSale4,045$20.81$84.2KForm 4
Feb 27, 2026Cvijic Christine MikailPresident and CFOSale4,800$25.11$120.5KForm 4
May 14, 2025Cobb StuartChief Scientific OfficerSale6,797$15.15$103.0KForm 4
Mar 13, 2025Cvijic Christine MikailPresident and CFOSale4,501$16.94$76.2KForm 4
Nov 26, 2024AKKARAJU SRINIVAS10% ownerBuy48,770$25.83$1.3MForm 4
Nov 22, 2024Cvijic Christine MikailPresident and CFOBuy24,000$20.48$491.5KForm 4
Nov 22, 2024McMinn RachelChief Executive OfficerBuy47,500$20.40$969.0KForm 4
Aug 17, 2023Baker Brothers Life Sciences LPDirectorBuy166,942$0.69$115.2KForm 4
Aug 16, 2023Baker Brothers Life Sciences LPDirectorBuy229,600$0.68$156.1KForm 4
Aug 15, 2023Baker Brothers Life Sciences LPDirectorBuy1,888,800$0.63$1.2MForm 4
Aug 11, 2023Smith Sean MichaelInterim CFO, Prin Acct OffSale1,323$0.62$820Form 4

All 25 trades can be filtered by person here when JavaScript is on.

From Forms 4 filed with the SEC.

Major holders

Investors who own more than 5% of the shares must tell the SEC: a 13D if they may seek to influence the company, a 13G if they hold passively.

Executive pay

What the company paid its top executives, from the summary compensation table in its proxy statement. Total pay includes salary, bonus, stock and option awards and other pay, as the company valued them.

YearExecutiveAgeSalaryTotal pay
2025Rachel McMinn$618.8K$3.2M88
2025Christine Mikail48$556.0K$2.8M88
2025Julie Jordan54$496.8K$1.5M88
2024Founder and Chief Executive Officer$595.0K$3.5M88
2024President and Chief Financial Officer$515.0K$3.4M88
2024Chief Medical Officer$460.0K$2.5M88

Held by these funds

The largest holders among investment managers' 13F reports for the quarter ending Jun 30, 2026, the top 25 of 100. Change is in shares since the quarter before.

FundSharesValueShare of the fundChange
Samsara Biocapital, LLC1,717,127$54.0M4.4%No change
Casdin Capital, LLC1,545,361$48.6M2.4%+125,000
Redmile Group, LLC1,412,199$44.4M3.0%−47,400
Rtw Investments, LP1,362,456$42.8M0.4%No change
EcoR1 Capital, LLC1,271,342$40.0M2.0%No change
BlackRock, Inc.1,057,983$33.3M0.0%+52,913
Baker Bros. Advisors LP991,691$31.2M0.2%No change
Balyasny Asset Management L.P.818,706$25.7M0.0%+100,317
Trails Edge Capital Partners, LP714,885$22.5M3.6%−67,902
Toronto Dominion Bank681,337$21.4M0.0%No change
Vanguard Capital Management LLC536,525$16.9M0.0%+21,798
Jennison Associates LLC518,582$16.3M0.0%−9,103
Citadel Advisors LLC408,982$12.9M0.0%+21,951
Great Point Partners LLC352,180$11.1M2.6%−147,820
ArrowMark Colorado Holdings LLC342,453$10.8M0.4%−182,343
Fmr LLC295,561$9.3M0.0%−87,086
Geode Capital Management, LLC293,182$9.2M0.0%+14,179
State Street Corp224,322$7.1M0.0%−88,683
Franklin Resources Inc204,280$6.4M0.0%New
Morgan Stanley191,724$6.0M0.0%−93,515
UBS Group AG171,289$5.4M0.0%−14,390
Blair William & Co/il146,905$4.6M0.0%−21,313
Driehaus Capital Management LLC143,528$4.5M0.0%+110,763
Point72 Asset Management, L.P.141,920$4.5M0.0%New
Moore Capital Management, LP141,636$4.5M0.1%New

13F reports cover disclosed long positions in U.S. stocks only.

SEC filer profile

How Neurogene Inc. is registered with the Securities and Exchange Commission, from its EDGAR record and the cover page of its latest annual report.

  • Name on EDGARNeurogene Inc.1
  • CIK0001404644
  • Employer ID (EIN)98-05425933
  • Filer statusNon-accelerated filer not an emerging growth company; a smaller reporting company; not a shell company3
  • Incorporated inDelaware3
  • Fiscal year endsDecember 311
  • Industry code (SIC)2834 Pharmaceutical Preparations1
  • SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance1
  • Business address535 W 24Th Street, 5Th Floor, New York, NY 100111
  • Phone(877) 237-50201
  • Shares outstanding20,011,382 as of Aug 7, 202611
  • Public float$146.0M as of Jun 30, 20253
  • AuditorDeloitte & Touche LLP, Morristown, NJ PCAOB ID 34; no auditor attestation of internal control over financial reporting3
  • On EDGAR sinceJun 25, 20071
  • Filings750 in all: 4 (232), 8-K (148), 3 (44), 10-Q (38), SC 13G/A (38), SC 13G (36), SC 13D/A (17), CORRESP (14)1
    All 48 form types

    S-8 (14), 424B5 (13), DEFA14A (13), SCHEDULE 13G/A (13), 10-K (12), DEF 14A (12), EFFECT (11), UPLOAD (10), 144 (8), S-3 (7), 4/A (6), 425 (6), ARS (5), D (5), SCHEDULE 13G (5), 8-K/A (4), SC 13D (4), 424B3 (3), 5 (3), S-4/A (3), 3/A (2), CT ORDER (2), PRE 14A (2), REGDEX (2), S-1 (2), SEC STAFF LETTER (2), 10-K/A (1), 10-Q/A (1), 424B4 (1), 8-A12B (1), CERTNAS (1), DRS (1), DRS/A (1), DRSLTR (1), NT 10-Q (1), POS AM (1), POS EX (1), REGDEX/A (1), S-1/A (1), S-4 (1)

  • Latest filingsAnnual report (10-K) Mar 24, 2026
    Quarterly report (10-Q) Aug 11, 2026
    Proxy statement (DEF 14A) Apr 16, 2026
    Latest 8-K (8-K) Aug 11, 2026

Securities registered for trading

ClassSymbolExchange
Common stock, par value $0.000001 per shareNGNEThe Nasdaq Global Market

From the cover page of the 10-K filed Mar 24, 2026 (Section 12(b) of the Exchange Act).3

Risk factors and legal proceedings

The risks Neurogene Inc. names in Item 1A of its 10-K filed Mar 24, 2026: 73 risks under these headings, as written.3

  • Risks Related to Our Limited Operating History, Financial Position and Capital Requirements
  • We have a limited operating history, have not completed any clinical trials, and have no products approved for commercial sale, and our results may vary from quarter to quarter.
  • We will require substantial additional capital to finance our operations in the future. If we are unable to raise such capital when needed, or on acceptable terms, we may be forced to delay, reduce or eliminate clinical trials, product development programs or future commercialization efforts.
  • We have incurred significant losses since inception, expect to incur significant losses for the foreseeable future and may not be able to achieve or sustain profitability in the future. We have no products for sale, have not generated any product revenue and may never generate product revenue or become profitable.
  • Risks Related to Discovery, Development and Commercialization
  • We are substantially dependent on the success of our most advanced product candidate, NGN-401, and our clinical trial of NGN-401 may not be successful.
  • We face competition from entities that have developed or may develop programs for the diseases we plan to address with NGN-401 and other product candidates in development.
  • NGN-401 is in clinical development and our preclinical programs are in early stages of development and may fail in development or suffer delays that materially and adversely affect their commercial viability. If we are unable to complete development of, or commercialize, our product candidates, or experience significant delays in doing so, our business will be materially harmed.
  • We currently utilize adeno-associated virus serotype 9 (“AAV9”) capsid for delivery of therapeutic transgenes to deliver our product candidates, which may limit the safety, purity, and efficacy or potency of such product candidates.
  • Participants in our clinical trials may experience undesirable side effects, which could cause delays or prevent regulatory approval of our product candidates, limit the commercial potential or create significant negative consequences to our development plans, even if such side effects are ultimately determined not to be attributable or possibly attributable to our product candidates.
  • We intend to identify and develop novel gene therapy product candidates, which makes it difficult to predict the time, cost and potential success of product candidate development.
  • The disorders we seek to treat have low prevalence and it may be difficult to identify and enroll patients with these disorders. If we experience delays or difficulties in the enrollment and/or maintenance of patients in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
The other 62 risks
  • We may not be successful in identifying and advancing a strategy to continue the development of NGN-101 for CLN5 Batten disease, and in the meantime, may incur additional costs as we continue the post-dosing phases of our clinical trial for NGN-101.
  • Our programs are focused on the development of therapeutics for patients with neurological diseases, which is a rapidly evolving area of science, and the approach we are taking to discover and develop product candidates is novel and may never lead to approved or marketable products.
  • If we do not achieve our projected development goals in the timeframes we announce and expect, the commercialization of NGN-401 or any other product candidates may be delayed and, as a result, our stock price may decline.
  • Preclinical and clinical development involves a lengthy and expensive process that is subject to delays and uncertain outcomes, and results of earlier studies and trials may not be predictive of future clinical trial results. If our preclinical studies and clinical trials are not sufficient to support regulatory approval of any of our product candidates, we may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development of such product candidate.
  • Preliminary, “topline” or interim data from our preclinical studies and clinical trials that we announce or publish from time to time may change as more patient data becomes available and are subject to audit and verification procedures.
  • Our current or future clinical trials may reveal significant adverse events or undesirable side effects not seen in our preclinical studies and may result in a safety profile that could halt clinical development, inhibit regulatory approval or limit commercial potential or market acceptance of NGN-401 or any other product candidates or result in potential product liability claims.
  • We may expend our limited resources to pursue a particular product candidate, such as NGN-401, and fail to capitalize on candidates that may be more profitable or for which there is a greater likelihood of success.
  • Even if regulatory approval is obtained, any approved products resulting from NGN-401 or any other product candidate may not achieve adequate market acceptance among clinicians, patients, healthcare third-party payors and others in the medical community necessary for commercial success and we may not generate any future revenue from the sale or licensing of such products.
  • We have never commercialized a product candidate and may lack the necessary expertise, personnel and resources to successfully commercialize a product candidate on our own or together with suitable collaborators.
  • We have never completed any late-stage clinical trials and may not be able to file an IND application or other applications for regulatory approval to commence additional clinical trials on the timelines we expect. Even if we are able to complete such trials, the FDA or comparable foreign regulatory authorities may not permit us to proceed or could suspend or terminate any such trial after it has been initiated.
  • Risks Related to Manufacturing
  • Gene therapies are novel, complex and difficult to manufacture. We could experience manufacturing problems that result in delays in the development or commercialization of our product candidates or otherwise harm our business.
  • We and our contract manufacturers for AAV9 are subject to significant regulation with respect to manufacturing of our products. The third-party manufacturing facilities on which we rely, our in-house manufacturing facility, and any manufacturing facility that we may have in the future, may have limited capacity or fail to meet the applicable stringent regulatory requirements.
  • We depend on third-party suppliers for materials used in the manufacture of our product candidates, and the loss of these third-party suppliers or their inability to supply us with adequate materials could harm our business.
  • Delays in developing our manufacturing capabilities or failure to achieve operating efficiencies from such capabilities may require us to devote additional resources and management time to manufacturing operations for future product candidates and may delay our product development timelines.
  • Any contamination or interruption in our manufacturing process, shortages of raw materials or failure of our suppliers to deliver necessary components could result in delays in our clinical development or marketing schedules.
  • We may not be able to successfully manufacture our product candidates in sufficient quality and quantity, which would delay or prevent us from developing our product candidates and commercializing resulting approved products, if any.
  • Changes in methods of product candidate manufacturing or formulation may result in additional costs or delay.
  • Risks Related to Our Reliance on Third Parties
  • We currently rely, and intend in the future to rely, on third parties to conduct a significant portion of our preclinical studies and existing clinical trials and potential future clinical trials for product candidates, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials.
  • Risks Related to Our Business and Operations
  • In order to successfully implement our plans and strategies, we will need to grow the size of our organization and we may experience difficulties in managing this growth.
  • We are highly dependent on our key personnel and anticipate hiring new key personnel. If we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy.
  • Our future growth may depend, in part, on our ability to operate in foreign markets, where we would be subject to additional regulatory burdens and other risks and uncertainties.
  • Our employees, independent contractors, consultants, commercial collaborators, principal investigators, CROs, CDMOs, suppliers and vendors may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements.
  • Our systems, or those of any of our CROs, CDMOs, manufacturers, other contractors, third party service providers or consultants or potential future collaborators, may fail or suffer security or data privacy breaches or other unauthorized or improper access to, use of, or destruction of our proprietary or confidential data, employee data or personal data, which could result in additional costs, loss of revenue, significant liabilities, harm to our brand and material disruption of our operations.
  • We are subject to stringent and changing laws, regulations and standards, and contractual obligations relating to privacy, data protection, and data security. The actual or perceived failure to comply with such obligations could lead to government enforcement actions (which could include civil or criminal penalties), fines and sanctions, private litigation, injunctive restrictions on data processing and/or adverse publicity and could negatively affect our operating results and business.
  • If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
  • Our ability to utilize our net operating loss carryforwards and certain other tax attributes may be limited.
  • We may be subject to adverse legislative or regulatory tax changes that could negatively impact our financial condition.
  • We may acquire businesses or products, or form strategic alliances, in the future, and may not realize the benefits of such acquisitions.
  • We maintain our cash at financial institutions, at times in balances that exceed federally-insured limits. The failure of financial institutions could adversely affect our ability to pay our operational expenses or make other payments.
  • Risks Related to Intellectual Property
  • Our ability to protect our patents and other proprietary rights is uncertain, exposing us to the possible loss of competitive advantage.
  • We may not be successful in obtaining or maintaining necessary rights to product candidates through acquisitions and in-licenses.
  • Certain of our current product candidates and research programs are licensed from or based upon licenses from a third party and are field limited to certain indications. If these license agreements are terminated or interpreted to narrow our rights, our ability to advance our current product candidates or develop new product candidates based on these technologies will be materially adversely affected.
  • If we fail to comply with our obligations in any agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
  • We may be subject to patent infringement claims or may need to file claims to protect our intellectual property, which could result in substantial costs, liability and diversion of resources, and prevent or delay us from commercializing potential products.
  • We may be subject to claims that we have wrongfully hired an employee from a competitor or that employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties.
  • Changes to patent laws in the United States and other jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our products.
  • Obtaining and maintaining patent protection depends on compliance with various procedural, document submissions, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
  • We may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent, which might adversely affect our ability to develop and market our products.
  • We may become subject to claims challenging the inventorship or ownership of our patents and other intellectual property.
  • Patent terms may be inadequate to protect our competitive position on our product candidates for an adequate amount of time.
  • Some intellectual property that we have in-licensed may have been discovered through government funded programs and thus may be subject to federal regulations such as “march-in” rights, certain reporting requirements and a preference for U.S.-based companies. Compliance with such regulations may limit our exclusive rights, and limit our ability to contract with non-U.S. manufacturers.
  • Risks Related to Government Regulation
  • The regulatory approval processes of the FDA and other comparable foreign regulatory authorities are lengthy, time-consuming and inherently unpredictable. If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals for our product candidates, or if we determine that we are not willing or able to complete the regulatory approval process given the resources required to do so, we will not be able to commercialize, or will be delayed in commercializing, such product candidates, and our ability to generate revenue will be materially impaired.
  • Because gene therapy is novel and the regulatory landscape that governs any product candidates we may develop is rigorous, complex, uncertain and subject to change, we cannot predict the time and cost of obtaining regulatory approval, if received at all, for any product candidates we may develop.
  • The FDA has recently required additional and in some cases more stringent clinical trial designs and clinical trial data for gene therapy product candidates, even where companies believed they had previously established alignment with the FDA on their trial designs. If the FDA were to change its position regarding elements of our clinical trial design or the clinical data required to support approval of our product candidates, we may face additional expenses or challenges in gaining approval of such products.
  • Disruptions at the FDA and other governmental agencies and regulatory authorities could negatively affect the review of our regulatory submissions or impact our ability to access the public markets, which could negatively impact our business.
  • We may not be able to meet requirements for the chemistry, manufacturing and control of our product candidates.
  • We intend to deliver our product candidates via a drug delivery device that will have its own regulatory, development, supply and other risks.
  • We have in the past and may in the future conduct clinical trials for our product candidates at sites outside the United States, and the FDA may not accept data from trials conducted in such locations.
  • Our product candidates for which we intend to seek approval as biologics may face competition sooner than anticipated.
  • Even if we receive regulatory approval of NGN-401 or other product candidates, we will be subject to extensive ongoing regulatory obligations and continued regulatory review, which may result in significant additional expense and we may be subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our product candidates.
  • We may face difficulties from healthcare legislative reform measures.
  • Our business operations and current and future arrangements with investigators, healthcare professionals, consultants, third-party payors, patient organizations and customers will be subject to applicable healthcare regulatory laws, which could expose us to penalties.
  • Even if we are able to commercialize NGN-401 or other product candidates, due to unfavorable pricing regulations and/or third-party coverage and reimbursement policies, we may not be able to offer such products at competitive prices which would seriously harm our business.
  • We are subject to U.S. and certain foreign export and import controls, sanctions, embargoes, anti-corruption laws, and anti-money laundering laws and regulations. We can face criminal liability and other serious consequences for violations, which can harm our business.
  • Healthcare legislative, regulatory or policy reform measures may have a negative impact on our business and results of operations, and could prevent commercial success of our product candidates.
  • Governments outside the United States may impose strict price controls, which may adversely affect our revenue, if any.
  • While we have received Fast Track designation and Breakthrough Therapy designation for NGN-401 for the treatment of Rett syndrome and NGN-401 has been accepted into the FDA’s START Pilot Program and RMAT program and has received PRIME designation from the EMA, such designations may not lead to a faster development or regulatory review or approval process.
  • We may seek certain additional designations for our product candidates, including Priority Review designations by the FDA, however, even if we receive such designations, there is no guarantee that they would lead to faster development or regulatory review timelines or increase the likelihood of marketing approval for such product candidate.
  • We have received orphan drug designation for NGN-401 for the treatment of Rett syndrome and we may seek orphan drug designation for certain future product candidates, but we may be unable to obtain such designations or to maintain the benefits associated with orphan drug designation, including market exclusivity, which may cause our revenue, if any, to be reduced.
  • We have received Rare Pediatric Disease designation by the FDA for NGN-401 for the treatment of Rett syndrome. However, Rare Pediatric Disease designation for any of our product candidates does not guarantee that the BLA for the product will qualify for a priority review voucher upon approval, and it does not lead to a faster development or regulatory review process, or increase the likelihood that our product candidates will receive marketing approval.
  • General Risk Factors
  • Our estimates of market opportunity and forecasts of market growth may prove to be inaccurate, and even if the markets in which we compete achieve the forecasted growth, our business may not grow at similar rates, or at all.
  • We may become exposed to costly and damaging liability claims, either when testing a product candidate in the clinical or at the commercial stage, and our product liability insurance may not cover all damages from such claims.

Legal proceedings

We may from time to time be named as a party to legal claims, actions and complaints, including matters involving employment, intellectual property or others. We are not presently a party to any legal proceedings that, in the opinion of our management, would reasonably be expected to have a material adverse effect on our business, financial condition, operating results or cash flows if determined adversely to us. Regardless of the outcome, litigation can have an adverse impact on us because of defense and settlement costs, diversion of management resources, and other factors.

The opening of Item 3, Legal Proceedings.3

Sources

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