EDIT on Nasdaq

Editas Medicine

Logo: Editas Medicine, Public domain, via Wikimedia Commons5

Discovery-phase pharmaceutical company

Editas Medicine, Inc., (formerly Gengine, Inc.), is a clinical-stage biotechnology company which is developing therapies for rare diseases based on CRISPR gene editing technology. Editas headquarters is located in Cambridge, Massachusetts and has facilities in Boulder, Colorado.3 Wikipedia text CC BY-SA 4.0

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Last updated Oct 21, 2026.

Overview

Jan 1, 2013Founded1

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  • TickerEDIT on Nasdaq1
  • Industrypharmaceutical industry1
  • HeadquartersCambridge1
  • Websitewww.editasmedicine.com
  • Legal formpublic company1
  • Incorporated inDE6
  • Business address11 Hurley St., Cambridge, Ma 021416
  • Filer categoryNon-accelerated filer, Smaller reporting company6
  • LEI5299000AON4Q9NT2JE424
  • StatusActive4
  • LEI statusLapsed (not renewed)4
  • Registered addressc/o THE CORPORATION TRUST COMPANY, CORPORATION TRUST CENTER 1209 ORANGE ST, 19801 Wilmington, United States4
  • Registered withDivision of Corporations, Department of State, 53927544

Editas Medicine, Inc., (formerly Gengine, Inc.), is a clinical-stage biotechnology company which is developing therapies for rare diseases based on CRISPR gene editing technology. Editas headquarters is located in Cambridge, Massachusetts and has facilities in Boulder, Colorado.3

Financials

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

Key numbers, fiscal 2025

-394.9%Operating margin8
-395.0%Net margin8
25.4%Revenue growth, 20258
-586.6%Return on equity8
1.96×Debt to equity8
-$165.8MFree cash flow8

Where the money goes

Where each dollar of revenue went, fiscal 2025

  • Research and development100¢$90.0M

Out of $40.5M in revenue.8 The company lost money this year.

Financial statements

Revenue91011128$40.5M in 2025+97% since 2019
0$25.0M$50.0M$75.0M$100.0M2019: $20.5M$20.5M20192020: $90.7M$90.7M2020+342%2021: $25.5M$25.5M2021−72%2022: $19.7M$19.7M2022−23%2023: $78.1M$78.1M2023+296%2024: $32.3M$32.3M2024−59%2025: $40.5M$40.5M2025+25%
Operating income91011128-$160.0M in 2025+14% since 2019
-$300.0M-$200.0M-$100.0M02019: -$140.9M-$140.9M20192020: -$134.8M-$134.8M2020−4%2021: -$193.1M-$193.1M2021+43%2022: -$225.9M-$225.9M2022+17%2023: -$169.2M-$169.2M2023−25%2024: -$251.2M-$251.2M2024+48%2025: -$160.0M-$160.0M2025−36%
Net income91011128-$160.1M in 2025+20% since 2019
-$300.0M-$200.0M-$100.0M02019: -$133.7M-$133.7M20192020: -$116.0M-$116.0M2020−13%2021: -$192.5M-$192.5M2021+66%2022: -$220.4M-$220.4M2022+15%2023: -$153.2M-$153.2M2023−30%2024: -$237.1M-$237.1M2024+55%2025: -$160.1M-$160.1M2025−32%

Every line of the income statement as filed: the last 2 fiscal years, latest first.8

Period ending2025-12-312024-12-31
Collaboration and other research and development revenues$40.5M$32.3M
Research and development$90.0M$199.2M
General and administrative$49.9M$72.0M
Restructuring and impairment charges$60.7M$12.2M
Total operating expenses$200.5M$283.5M
Operating loss-$160.0M-$251.2M
Interest expense related to sale of future revenues-$6.2M-$2.2M
Interest income, net$8.3M$16.3M
Other expense, net-$2.2M-$3.0K
Total other income (expense), net-$50.0K$14.1M
Net loss-$160.1M-$237.1M
Net loss per share, basic (in dollars per share)-$1.80-$2.88
Net loss per share, diluted (in dollars per share)-$1.80-$2.88
Weighted-average common shares outstanding, basic (in shares)88.7M shares82.3M shares
Weighted-average common shares outstanding, diluted (in shares)88.7M shares82.3M shares

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Corporate structure

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

Major holders of Editas Medicine

  • Editas Medicine Cambridge, Massachusetts, United States

Built from parent and subsidiary links from GLEIF, the Global Legal Entity Identifier Foundation (CC0); major holders from Schedules 13D and 13G. Each entity in the chart links to its source.

What it owns

What Editas Medicine 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$31.9M8 Dec 31, 2025$46.1M8 Dec 31, 2024−30.9%
Accumulated depreciation$28.4M8 Dec 31, 2025$31.6M8 Dec 31, 2024−10.4%
Property and equipment, net$3.5M8 Dec 31, 2025$14.5M8 Dec 31, 2024−75.6%
Operating-lease assets (leased space)$16.1M8 Dec 31, 2025$32.6M8 Dec 31, 2024−50.5%

Investments

ItemLatestA year beforeChange
Cash and cash equivalents$146.6M8 Dec 31, 2025$131.5M8 Dec 31, 2024+11.5%
Marketable securities, current$08 Dec 31, 2025$138.4M8 Dec 31, 2024−100.0%
Marketable securities, non-current$012 Dec 31, 2024$104.0M12 Dec 31, 2023−100.0%
Debt securities available for sale$146.6M8 Dec 31, 2025$269.9M8 Dec 31, 2024−45.7%

Properties, in its own words

We lease 59,783 square feet of office and laboratory space in Cambridge, Massachusetts under a lease that expires in October 2028. We believe that our facilities are sufficient to meet our current needs and that suitable additional space will be available as and when needed.

Item 2 of the 10-K filed Mar 9, 2026.16

The story

Editas Medicine was originally founded with the name "Gengine, Inc." in September 2013 by Feng Zhang of the Broad Institute, Jennifer Doudna of the University of California, Berkeley, and George Church, David Liu, and J. Keith Joung of Harvard University, with funding from Third Rock Ventures, Polaris Partners and Flagship Ventures. The name was changed to the current "Editas Medicine" two months later. Doudna quit in June 2014 over legal differences concerning intellectual property of Cas9.

In August 2015, the company raised $120 million in Series B funding from Bill Gates and 13 other investors. it went public on 2 February 2016, via a $94 million initial public offering.

Editas entered into a strategic collaboration with Juno Therapeutics in 2015 to create a chimeric antigen receptor and high-affinity T cell receptor therapeutics to the end of developing cancer therapeutics. Juno was later acquired by Celgene, which was in turn acquired by Bristol Myers Squibb.

The company announced in 2015 that it was planning a clinical trial using CRISPR gene editing techniques to treat Leber congenital amaurosis type 10 (LCA10), a rare genetic illness that causes blindness. On 30 November 2018, the FDA gave permission to start the trials, under the investigational name EDIT-101 (also known as AGN-151587). In September 2021, a statement from Editas claimed that preliminary results from clinical trials were promising and support clinical benefits of EDIT-101 treatment.

In March 2020, Editas, in partnership with Allergan, was the first to use CRISPR to try to edit DNA inside a person's body (in vivo). As part of the clinical trial, a patient who was nearly blind as a result of Leber's congenital amaurosis received an intravitreal injection containing a harmless virus carrying CRISPR gene-editing instructions. Five months later, Editas reworked its deal with Allergan's owner AbbVie and regained full rights to their range of eye disease treatment therapies, including EDIT-101 for the treatment of LCA10.

In 2019, the company was building new chemistry facilities in Boulder, Colorado.

Katrine Bosley was CEO until 2019, when she was replaced by board member Cynthia Collins. Collins was replaced in 2021 by James Mullen, who had been board chairman. Gilmore O'Neill, former CMO of Sarepta Therapeutics, became CEO on June 1, 2022, with Mullen staying on as executive chairman of the board.

On December 12, 2024, Editas ended the development of reni-cel and laid off 65% of its employees.

From Wikipedia’s article on Editas Medicine, shared under CC BY-SA 4.0.7

Leadership

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

Board members and other officers (27)

Products and brands

Editas works with two different CRISPR nucleases, Cas9 and Cas12a.

EDIT-101 is a CRISPR based gene therapy for treatment of Leber congenital amaurosis, currently in clinical trials.

EDIT-301 is an experimental potential treatment utilizing the firm's CAS 12a editing technology for sickle cell disease and beta-thalassemia. In 2019 the firm reported early success in research on the drug;. In December 2020, it filed an IND application for treatment of sickle cell disease. In January 2021, it said it had received clearance from the FDA for phase 1 safety studies.

From Wikipedia’s article on Editas Medicine, shared under CC BY-SA 4.0.7

Timeline

  1. Oct 8, 20268-K Regulation FD disclosure; Other events52
  2. Aug 5, 20268-K Results of operations; Executive or director change53
  3. Jun 18, 20268-K Shareholder vote54
  4. May 26, 20268-K Regulation FD disclosure; Other events55
  5. May 26, 20268-K Other events56
  6. May 26, 20268-K Entered a material agreement; Other events57
  7. May 14, 20268-K Regulation FD disclosure; Other events58
  8. May 5, 20268-K Results of operations59
  9. Apr 7, 20268-K Change of auditor60
  10. Mar 27, 20268-K Regulation FD disclosure; Other events61
  11. Mar 9, 20268-K Results of operations62
  12. Nov 10, 20258-K Results of operations63
  13. Jun 6, 2022New chief executive Gilmore Neil O'Neill becomes CEO17
  14. Mar 4, 2021New chief executive James Mullen becomes CEO18
  15. Jun 14, 2019New chief executive Cynthia Collins becomes CEO19
  16. Feb 2, 2016New chief executive Katrine Bosley becomes CEO20
  17. Jan 1, 2013Founded Founded1

Calendar

Coming up

  1. Nov 9, 2026Reports results for the quarter ending Sep/2026 A year earlier it reported ($0.28) a share, on Nov 10, 2025.64

When it reported results

  1. Aug 5, 2026Reported quarterly or annual results53
  2. May 5, 2026Reported quarterly or annual results59
  3. Mar 9, 2026Reported quarterly or annual results62
  4. Nov 10, 2025Reported quarterly or annual results63
  5. Aug 12, 2025Reported quarterly or annual results65
  6. May 12, 2025Reported quarterly or annual results66
  7. Mar 5, 2025Reported quarterly or annual results67
  8. Jan 13, 2025Reported quarterly or annual results68

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

Filings

310 filings to the SEC since October 2021: 5 annual reports, 15 quarterly reports, 61 major events, 145 insider forms, 30 large holders, 54 other. The latest 20:

  1. Oct 8, 20268-K EDGAR
  2. Sep 9, 20264 EDGAR
  3. Sep 9, 20263 EDGAR
  4. Sep 9, 2026S-8 EDGAR
  5. Sep 8, 2026EFFECT EDGAR
  6. Sep 4, 20264 EDGAR
  7. Sep 4, 20264 EDGAR
  8. Sep 4, 20264 EDGAR
  9. Aug 31, 2026144 EDGAR
  10. Aug 31, 2026144 EDGAR
  11. Aug 31, 2026S-3 EDGAR
  12. Aug 14, 2026SCHEDULE 13G EDGAR
  13. Aug 7, 20264 EDGAR
  14. Aug 7, 20263 EDGAR
  15. Aug 5, 20264 EDGAR
  16. Aug 5, 202610-Q EDGAR
  17. Aug 5, 20268-K EDGAR
  18. Jul 29, 2026SCHEDULE 13G/A EDGAR
  19. Jul 29, 20264 EDGAR
  20. Jul 21, 2026144 EDGAR

The 310 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

75 open-market trades by officers, directors and large owners since October 2021: 1 buys and 74 sales. Grants, option exercises and gifts are left out.

DateWhoTradeSharesPriceValueFiling
Sep 3, 2026Parison AmySVP, Chief Financial OfficerSale453$3.04$1.4KForm 4
Sep 3, 2026Burkly LindaEVP, CHIEF SCIENTIFIC OFFICERSale709$3.04$2.2KForm 4
Sep 3, 2026O'Neill Gilmore NeilCEOSale5,124$3.04$15.6KForm 4
Aug 3, 2026Parison AmySVP, Chief Financial OfficerSale678$2.58$1.7KForm 4
Jul 28, 2026Burkly LindaEVP, CHIEF SCIENTIFIC OFFICERSale4,928$2.67$13.2KForm 4
Jun 3, 2026Parison AmySVP, Chief Financial OfficerSale464$2.70$1.3KForm 4
Jun 3, 2026O'Neill Gilmore NeilCEOSale15,380$2.70$41.5KForm 4
Jun 3, 2026Burkly LindaEVP, CHIEF SCIENTIFIC OFFICERSale731$2.70$2.0KForm 4
Mar 3, 2026O'Neill Gilmore NeilCEOSale5,394$2.02$10.9KForm 4
Mar 3, 2026Parison AmySVP, Chief Financial OfficerSale474$2.02$957Form 4
Mar 3, 2026Burkly LindaEVP, CHIEF SCIENTIFIC OFFICERSale749$2.02$1.5KForm 4
Dec 3, 2025Burkly LindaEVP, CHIEF SCIENTIFIC OFFICERSale713$2.15$1.5KForm 4
Dec 3, 2025O'Neill Gilmore NeilCEOSale5,603$2.15$12.0KForm 4
Dec 3, 2025Parison AmySVP, Chief Financial OfficerSale461$2.15$991Form 4
Sep 3, 2025Burkly LindaEVP, CHIEF SCIENTIFIC OFFICERSale710$2.60$1.8KForm 4
Sep 3, 2025Parison AmySVP, Chief Financial OfficerSale458$2.60$1.2KForm 4
Sep 3, 2025O'Neill Gilmore NeilCEOSale5,592$2.60$14.5KForm 4
Aug 7, 2025Parison AmySVP, Chief Financial OfficerSale679$2.58$1.8KForm 4
Jul 31, 2025Burkly LindaEVP, CHIEF SCIENTIFIC OFFICERSale5,121$2.49$12.8KForm 4
Jun 3, 2025Parison AmySVP, Chief Financial OfficerSale446$1.72$767Form 4

All 75 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
2025Gilmore O’NeillM.B., M.M.Sc.$686.6K$2.4M80
2025Linda C. BurklyPh.D.$517.2K$1.2M80
2025Amy Parison$394.2K$821.7K80
2024President and Chief Executive Officer$667.1K$5.1M80

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
TCG Crossover Management, LLC11,111,111$36.0M0.9%New
BlackRock, Inc.10,589,990$34.3M0.0%+1,326,678
Ra Capital Management, L.P.8,889,000$28.8M0.3%New
Commodore Capital LP8,889,000$28.8M1.7%New
ADAR1 Capital Management, LLC8,889,000$28.8M1.4%New
Vanguard Capital Management LLC6,141,336$19.9M0.0%+1,936,967
Paradigm Biocapital Advisors LP5,586,625$18.1M0.3%New
Opaleye Management Inc.3,861,930$12.5M1.1%+1,934,933
Vanguard Portfolio Management LLC3,598,339$11.7M0.0%+449,769
Woodline Partners LP3,555,497$11.5M0.0%New
Vivo Capital, LLC3,555,250$11.5M1.1%New
GordonMD Global Investments LP3,127,000$10.1M4.9%New
Goldman Sachs Group Inc2,614,036$8.5M0.0%+2,055,459
Geode Capital Management, LLC2,569,072$8.3M0.0%+257,283
Renaissance Technologies LLC2,468,919$8.0M0.0%−1,503,241
Logos Global Management LP2,222,000$7.2M0.3%New
State Street Corp2,191,803$7.1M0.0%+115,681
Marshall Wace, Llp2,037,287$6.6M0.0%+1,826,979
D. E. Shaw & Co., Inc.1,901,002$6.2M0.0%−42,891
Jpmorgan Chase & Co1,862,494$6.0M0.0%−84,890
Nantahala Capital Management, LLC1,705,400$5.5M0.2%New
Aqr Capital Management LLC1,572,242$5.1M0.0%+201,745
Two Sigma Investments, LP1,332,976$4.3M0.0%−216,087
Seven Fleet Capital Management LP1,330,500$4.3M0.7%New
Raymond James Financial Inc1,318,947$4.3M0.0%+25,099

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

Members of Congress who traded it

11 trades disclosed by members of Congress. “Vs S&P since” is the stock’s change since the trade minus the S&P 500’s.

DateMemberTradeAmountVs S&P sinceFiling
Apr 10, 2023Daniel S. GoldmanDemocratic, HouseSale$1,001 - $15,000+153.0%Report
Apr 14, 2021Donald S. Beyer, Jr.Democratic, HouseSale$1,001 - $15,000+181.9%Report
Mar 2, 2021Donald S. Beyer, Jr.Democratic, HouseSale$1,001 - $15,000+194.5%Report
Nov 17, 2020Donald S. Beyer, Jr.Democratic, HouseBuy$1,001 - $15,000−205.8%Report
May 1, 2020Donald S. Beyer, Jr.Democratic, HouseSale$1,001 - $15,000+262.3%Report
Apr 27, 2020Donald S. Beyer, Jr.Democratic, HouseSale$1,001 - $15,000+259.6%Report
Jan 27, 2020Donald S. Beyer, Jr.Democratic, HouseBuy$1,001 - $15,000−230.2%Report
Jan 9, 2020Donald S. Beyer, Jr.Democratic, HouseBuy$1,001 - $15,000−228.6%Report
Dec 23, 2019Donald S. Beyer, Jr.Democratic, HouseBuy$1,001 - $15,000−233.0%Report
Jun 4, 2019Donna Shalala, HouseSale$1,001 - $15,000+263.8%Report
Mar 6, 2019Donna Shalala, HouseBuy$1,001 - $15,000−267.9%Report

Members report amounts as ranges, not exact figures. Returns are price-only (no dividends).

SEC filer profile

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

  • Name on EDGAREditas Medicine, Inc.2
  • CIK0001650664
  • Employer ID (EIN)46-409752816
  • Filer statusNon-accelerated filer not an emerging growth company; a smaller reporting company; not a shell company16
  • Incorporated inDelaware16
  • Fiscal year endsDecember 312
  • Industry code (SIC)2836 Biological Products, (No Diagnostic Substances)2
  • SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance2
  • Business address11 Hurley St., Cambridge, MA 021412
  • Phone617-401-90002
  • Shares outstanding153,580,035 as of Jul 31, 202614
  • Public float$191.4M as of Jun 30, 202516
  • AuditorErnst & Young LLP, Boston, Massachusetts PCAOB ID 42; no auditor attestation of internal control over financial reporting16
  • On EDGAR sinceAug 18, 20152
  • Filings726 in all: 4 (298), 8-K (131), 3 (45), SC 13G/A (43), 10-Q (32), 144 (23), CT ORDER (14), 424B5 (13)2
    All 42 form types

    DEFA14A (12), S-8 (12), SC 13G (12), 10-K (11), DEF 14A (10), 424B7 (8), CORRESP (5), EFFECT (5), S-3ASR (5), 4/A (4), SCHEDULE 13G (4), SCHEDULE 13G/A (4), SEC STAFF LETTER (4), ARS (3), S-3 (3), SC 13D/A (3), UPLOAD (3), DRS/A (2), DRSLTR (2), 10-Q/A (1), 424B1 (1), 8-A12B (1), 8-K/A (1), CERTNAS (1), D (1), DRS (1), POS AM (1), POSASR (1), PRE 14A (1), S-1 (1), S-1/A (1), S-3/A (1), S-3MEF (1), SC 13D (1)

  • Latest filingsAnnual report (10-K) Mar 9, 2026
    Quarterly report (10-Q) Aug 5, 2026
    Proxy statement (DEF 14A) Apr 28, 2026
    Latest 8-K (8-K) Oct 8, 2026

Securities registered for trading

ClassSymbolExchange
Common StockEDITThe Nasdaq Stock Market LLC

From the cover page of the 10-K filed Mar 9, 2026 (Section 12(b) of the Exchange Act).16

Risk factors and legal proceedings

The risks Editas Medicine names in Item 1A of its 10-K filed Mar 9, 2026: 73 risks under these headings, as written.16

  • Risks Related to Our Financial Position and Need for Additional Capital
  • We have incurred significant losses since inception. We expect to incur losses for the foreseeable future and may never achieve or maintain profitability.
  • We will need substantial additional funding. If we are unable to raise capital when needed, we would be forced to delay, reduce, or eliminate our research and product development programs or commercialization efforts.
  • Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
  • Our operating history may make it difficult for our stockholders to evaluate the success of our business to date and to assess our future viability.
  • We have never generated revenue from product sales and may never be profitable.
  • Unfavorable national or global economic conditions or political developments could adversely affect our business, financial condition or results of operations.
  • Risks Related to Discovery, Development, and Commercialization
  • We are dependent on the success of our lead product candidate, EDIT-401, which is in preclinical development. Development of product candidates may not be successful. If we are unable to commence and complete the clinical development of, obtain marketing approval for, or successfully commercialize EDIT-401, either alone or with a collaborator, or if we experience significant delays in doing so, our business would be substantially harmed.
  • We intend to identify and develop product candidates based on a relatively novel gene editing technology, which makes it difficult to predict the time and cost of product candidate development. Only one therapeutic product that utilizes gene editing technology has been approved in the United States or in Europe.
  • Regulatory requirements governing genetic medicines, and in particular any novel genetic medicines we may develop, have changed frequently and may continue to change in the future.
  • We may not be successful in our efforts to identify, develop, or commercialize potential product candidates.
The other 63 risks
  • The gene editing field is relatively new and is evolving rapidly. We are focusing our research and development efforts on CRISPR gene editing technology using Cas9 and Cas12a enzymes, but other gene editing technologies may be discovered that provide significant advantages over CRISPR/Cas9 or CRISPR/Cas12a, which could materially harm our business.
  • All of our product development programs are at the preclinical or research stage. Preclinical testing and clinical trials of product candidates, including EDIT-401, may not be successful. If we are unable to commercialize any product candidates we develop or experience significant delays in doing so, our business will be materially harmed.
  • If serious adverse events, undesirable side effects, or unexpected characteristics are identified during the development of any product candidates we may develop, we may need to abandon or limit our further clinical development of those product candidates.
  • If any of the product candidates we develop or the delivery modes we rely on cause undesirable side effects, it could delay or prevent their regulatory approval, limit the commercial potential, or result in significant negative consequences following any potential marketing approval.
  • We have not tested any of our proposed delivery modes, combined with our product candidates, in clinical trials and have not begun clinical trials in any of our current development programs.
  • Because we are developing product candidates for the treatment of diseases in which there is little clinical experience using new technologies, there is increased risk that the FDA, the EMA, or other regulatory authorities may not consider the endpoints of our future clinical trials to provide clinically meaningful results and that these results may be difficult to analyze.
  • We currently plan to conduct and may in the future conduct clinical trials for our product candidates outside the U.S., and the FDA and comparable foreign regulatory authorities may not accept data from such trials.
  • If clinical trials of any product candidates we identify and develop fail to demonstrate safety and efficacy to the satisfaction of regulatory authorities or do not otherwise produce positive results, we may incur additional costs or
  • experience delays in completing, or ultimately be unable to complete, the development and commercialization of such product candidates.
  • If we experience delays or difficulties in the enrollment of patients in clinical trials, our ability to conduct clinical trials on a timely basis or at all and receipt of necessary regulatory approvals could be delayed or prevented.
  • We may expend our limited resources to pursue a particular product candidate or indication and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success.
  • If we are unable to successfully identify patients who are likely to benefit from therapy with any medicines we develop, or experience significant delays in doing so, we may not realize the full commercial potential of any medicines we may develop.
  • Even if we complete the necessary clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize a product candidate we develop, and any such approval may be for a more narrow indication than we seek.
  • Even if any product candidates we develop receive marketing approval, they may fail to achieve the degree of market acceptance by physicians, patients, healthcare payors, and others in the medical community necessary for commercial success.
  • Adverse public perception of genomic medicines, and gene editing in particular, may negatively impact regulatory approval of, or demand for, our potential products.
  • If, in the future, we are unable to establish sales and marketing capabilities or enter into agreements with third parties to sell and market any of our product candidates, we may not be successful in commercializing those product candidates if and when they are approved.
  • We face significant competition in an environment of rapid technological change, and our competitors may achieve regulatory approval before us or develop therapies that are safer or more advanced or effective than ours.
  • If we are able to commercialize any product candidates, such products may become subject to unfavorable pricing regulations, third-party reimbursement practices, or healthcare reform initiatives, which would harm our business.
  • Due to the novel nature of our technology and the potential for some of our product candidates to offer therapeutic benefit in a single administration or limited number of administrations, we face uncertainty related to pricing and reimbursement for these product candidates.
  • Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of any medicines that we may develop.
  • If we or any contract manufacturers and suppliers we engage 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.
  • Genomic medicines are novel, and our product candidates can be complex and difficult to manufacture. We could experience production problems that result in delays in our development or commercialization programs, limit the supply of our products, or otherwise harm our business.
  • Risks Related to Our Dependence on Third Parties
  • We expect to depend on collaborations with third parties for the research, development, and commercialization of certain of the product candidates we develop or for development of certain of our research programs. If any such collaborations are not successful, we may not be able to capitalize on the market potential of those product candidates or research programs.
  • If we are not able to establish collaborations on commercially reasonable terms, we may have to alter our development and commercialization plans.
  • We expect to rely on third parties to conduct our future clinical trials and some aspects of our research and preclinical testing, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials, research, or testing.
  • We contract with third parties for the manufacture of materials for our research programs and preclinical studies and expect to do so in the future for clinical trials and for commercialization of any product candidates that we develop. This reliance on third parties increases the risk that we will not have sufficient quantities of such materials, product candidates, or any medicines that we may develop and commercialize, or that such supply will not be available to us at an acceptable cost, which could delay, prevent, or impair our development or commercialization efforts.
  • Risks Related to Our Intellectual Property
  • If we are unable to obtain and maintain patent protection for any products we develop and for our technology, or if the scope of the patent protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and our ability to successfully commercialize any of our product candidates, and our technology may be adversely affected.
  • Our rights to develop and commercialize our technology and product candidates are subject, in part, to the terms and conditions of licenses granted to us by others.
  • We may not be able to protect our intellectual property and proprietary rights throughout the world.
  • Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment, and other requirements imposed by government patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
  • If we fail to comply with our obligations in the agreements under which we 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 not be successful in obtaining necessary rights to any product candidates we develop through acquisitions and in-licenses.
  • Issued patents covering our technology and product candidates could be found invalid or unenforceable if challenged in court or before administrative bodies in the United States or abroad.
  • The intellectual property landscape around gene editing technology, including CRISPR, is highly dynamic, and third parties may initiate legal proceedings alleging that we are infringing, misappropriating, or otherwise violating their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business.
  • If we do not obtain patent term extension and data exclusivity for any product candidates we may develop, it could have a material adverse effect on our business.
  • We may be subject to claims that our employees, consultants, or advisors have wrongfully used or disclosed alleged trade secrets of their current or former employers or claims asserting ownership of what we regard as our own intellectual property.
  • If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
  • Risks Related to Regulatory Approval and Other Legal Compliance Matters
  • Even if we complete the necessary preclinical studies and clinical trials, the marketing approval process is expensive, time-consuming, and uncertain and may prevent us from obtaining approvals for the commercialization of any of our product candidates. If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals, we will not be able to commercialize, or will be delayed in commercializing, our product candidates, and our ability to generate revenue will be materially impaired.
  • We may seek certain designations for our product candidates, including Breakthrough Therapy, Fast Track and Priority Review designations in the U.S., and PRIME Designation in the EU, but we might not receive such designations, and even if we do, such designations may not lead to a faster development or regulatory review or approval process.
  • If approved, our product candidates that are licensed and regulated as biologics may face competition from biosimilars approved through an abbreviated regulatory pathway.
  • We may not be able to obtain orphan drug exclusivity for one or more of our product candidates, and even if we do, that exclusivity may not prevent the FDA or the EMA from approving other competing products.
  • Failure to obtain marketing approval in foreign jurisdictions would prevent any of our product candidates from being marketed in such jurisdictions, which, in turn, would materially impair our ability to generate revenue.
  • Even if we, or any collaborators we may have, obtain marketing approvals for any of our product candidates, the terms of approvals and ongoing regulation of our products could require the substantial expenditure of resources and may limit how we, or they, manufacture and market our products, which could materially impair our ability to generate revenue.
  • Disruptions at the FDA and other government agencies from funding cuts, personnel losses, regulatory reform, government shutdowns and other developments could hinder our ability to obtain guidance from the FDA regarding our clinical development programs and develop and secure approval of our product candidates in a timely manner, which would negatively impact our business.
  • Even if our product candidates receive regulatory approval, they will be subject to significant post-marketing regulatory requirements and oversight.
  • Our relationships with healthcare providers, physicians, and third-party payors will be subject to applicable anti-kickback, fraud and abuse, and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm, and diminished profits and future earnings.
  • Enacted and future legislation may increase the difficulty and cost for us and any future collaborators to obtain marketing approval of and commercialize our product candidates and affect the prices we, or they, may obtain.
  • The prices of prescription pharmaceuticals in the United States and foreign jurisdictions is subject to considerable legislative and executive actions and could impact the prices we obtain for our products, if and when licensed.
  • Our employees, principal investigators, consultants, and commercial partners may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading.
  • Laws and regulations governing any international operations we may have in the future may preclude us from developing, manufacturing and selling certain product candidates outside of the United States and require us to develop and implement costly compliance programs.
  • We are subject to stringent privacy laws, information security laws, regulations, policies and contractual obligations related to data privacy and security and changes in such laws, regulations, policies, contractual obligations and failure to comply with such requirements could subject us to significant fines and penalties, which may have a material adverse effect on our business, financial condition or results of operations.
  • Risks Related to Employee Matters, Managing Growth, Public Health and Information Technology
  • Our future success depends on our ability to attract and retain key executives and to attract, retain, and motivate qualified personnel.
  • Security breaches and other disruptions to our information technology structure could compromise our information, disrupt our business and expose us to liability, which would cause our business and reputation to suffer.
  • Risks Related to Our Common Stock
  • The market price of our common stock has been, and is likely to remain, volatile.
  • If securities analysts do not publish research or reports about our business or if they publish negative evaluations of our stock, the price of our stock and trading volume could decline.
  • Future sales and issuances of our common stock or rights to purchase common stock, including pursuant to our equity incentive plans and “at-the-market” offerings, could result in additional dilution of the percentage ownership of stockholders and could cause our stock price to fall.
  • We incur costs as a result of operating as a public company, and our management is required to devote substantial time to compliance initiatives and corporate governance practices.
  • We have broad discretion in the use of our cash reserves and may not use them effectively, including that we may be exposed to liquidity issues and other systemic financial risks at the financial institutions holding our cash and cash equivalents.
  • We do not expect to pay any dividends for the foreseeable future. Accordingly, stockholders must rely on capital appreciation, if any, for any return on their investments.
  • Provisions in our restated certificate of incorporation and amended and restated bylaws or Delaware law might discourage, delay, or prevent a change in control of our company or changes in our management and, therefore, depress the trading price of our common stock.
  • Our restated certificate of incorporation designates the state courts in the State of Delaware or, if no state court located within the State of Delaware has jurisdiction, the federal court for the District of Delaware, as the sole and exclusive forum for certain types of actions and proceedings that may be initiated by our stockholders, which could discourage lawsuits against the company and our directors and officers.
  • We are a “smaller reporting company” within the meaning of the Securities Exchange Act of 1934, and if we decide to take advantage of certain exemptions from various reporting requirements applicable to smaller reporting companies, our common stock could be less attractive to investors.

Legal proceedings

From time to time, we may become involved in litigation or other legal proceedings relating to claims arising from the ordinary course of business. There can be no assurance that any proceedings that result from these third-party actions will be resolved in our favor. In addition, if they are not resolved in our favor, there can be no assurance that the result will not have a material adverse effect on our business, financial condition, results of operations, or prospects. Certain of our intellectual property rights, including ones licensed to us under our licensing agreements, are subject to, and from time to time may be subject to, priority and validity disputes. For additional information regarding these matters, see Part I, Item 1A. “Risk Factors—Risks Related to Our Intellectual Property” of this Annual Report on Form 10-K. Regardless of outcome, litigation or other legal proceedings 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.16

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