CRISPR Therapeutics (NASDAQ: CRSP) posts Q1 2026 loss and sells $600M converts
CRISPR Therapeutics’ Q1 2026 report shows it remains a late‑stage R&D company with substantial cash and a larger capital base. Total revenue was modest at $1.5 million, while operating expenses reached $131.7 million, driven mainly by $68.6 million in research and development and $45.9 million of collaboration expense under its Vertex hemoglobinopathy agreements.
The company reported a net loss of $122.9 million, slightly improved from $136.0 million a year earlier, with basic and diluted loss per share of $1.28 on 96.1 million weighted‑average shares. Operating cash outflow was $108.9 million, partly offset by strong financing inflows.
Liquidity remains a key strength. As of March 31 2026, cash, cash equivalents and marketable securities totaled about $2.44 billion, within total assets of $2.73 billion. During the quarter CRISPR issued $600 million of Convertible Senior Notes due 2031 at a 1.7308% coupon, creating $585.5 million of long‑term debt and net proceeds of about $585.4 million. The added capital supports continued investment across its CASGEVY hemoglobinopathy franchise, in vivo liver programs such as CTX310, siRNA collaboration asset CTX611 and next‑generation CAR‑T candidate zugo‑cel.
Positive
- None.
Negative
- None.
Insights
CRISPR Therapeutics adds low‑coupon debt, extends cash runway while continuing heavy R&D spend.
CRISPR Therapeutics reported a Q1 2026 net loss of $122.9 million on minimal revenue of $1.5 million, typical for a clinical‑stage biotech. Operating expenses of $131.7 million reflect broad investment in CASGEVY, in vivo liver editing, CAR‑T, siRNA and regenerative medicine programs.
The balance sheet is strong. Cash, cash equivalents and marketable securities totaled about $2.44 billion as of March 31 2026. The company issued $600 million of Convertible Senior Notes due 2031 at a 1.7308% coupon, adding long‑term debt of $585.5 million net of issuance costs while preserving equity.
Key dependencies are continued access to capital and successful execution of partnered programs, notably with Vertex and Sirius. Collaboration expense of $45.9 million with Vertex and siRNA‑related costs of $2.0 million show reliance on these alliances. Future disclosures on CASGEVY profitability, use of the new note proceeds and progress in CTX310, CTX611 and zugo‑cel will shape how this spending translates into long‑term value.
Key Figures
Key Terms
Convertible Senior Notes financial
collaboration expense, net financial
regenerative medicine medical
SyNTase editing technical
regenerative medicine advanced therapy designation regulatory
collaborative arrangements financial
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the quarterly period ended
or
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the transition period from to .
Commission File Number:
(Exact name of registrant as specified in its charter)
Not Applicable |
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(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer Identification No.) |
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Not Applicable |
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(Address of principal executive offices) |
(Zip Code) |
+
(Registrant’s telephone number, including area code)
Securities registered pursuant to Section 12(b) of the Act:
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered |
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or emerging growth company. See definition of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
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Accelerated filer |
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Non-accelerated filer |
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Smaller reporting company |
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Emerging growth company |
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No
As of April 30, 2026, there were
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Throughout this Quarterly Report on Form 10-Q, the “Company,” “CRISPR,” “CRISPR Therapeutics,” “we,” “us,” and “our,” except where the context requires otherwise, refer to CRISPR Therapeutics AG and its consolidated subsidiaries; “our board of directors” refers to the board of directors of CRISPR Therapeutics AG; and we generally refer to CASGEVY® (exagamglogene autotemcel [exa-cel]), as “CASGEVY”.
“CRISPR Therapeutics®” standard character mark and design logo, “CRISPRX™,” “CRISPR TX™,” “CTX213™,” “CTX310®,” “CTX321™,” “CTX340™,” “CTX460™,” “CTX611™”and “SyNTase™” are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated, and Vertex Pharmaceuticals Incorporated is the manufacturer and exclusive license holder of CASGEVY. All other trademarks and registered trademarks contained in this Quarterly Report on Form 10-Q are the property of their respective owners. Solely for convenience, trademarks, service marks and trade names referred to in this Quarterly Report on Form 10-Q may appear without the ® or ™ symbols and any such omission is not intended to indicate waiver of any such rights.
FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q contains “forward-looking statements” that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Quarterly Report on Form 10-Q are forward-looking statements. These statements are often identified by the use of words such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “potential,” “will,” “would” or the negative or plural of these words or similar expressions or variations, although not all forward-looking statements contain these identifying words. Forward-looking statements in this Quarterly Report on Form 10-Q include, but are not limited to, statements about:
Any forward-looking statements in this Quarterly Report on Form 10-Q reflect our current views with respect to future events or to our future financial performance and involve known and unknown risks, uncertainties and assumptions that could cause our actual results and the timing of certain events to differ materially from future results expressed or implied by the forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those identified herein, and those discussed in the section titled “Risk Factors,” set forth in Part II, Item 1A of this Quarterly Report on Form 10-Q, if any, our Annual Report on Form 10-K filed with the Securities and Exchange Commission, or SEC, on February 12, 2026, and in other SEC filings. You should
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not rely upon forward-looking statements as predictions of future events. Such forward-looking statements speak only as of the date of this report. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures or investments we may make or enter into.
This Quarterly Report on Form 10-Q may also contain information regarding our industry, our business and the markets for certain of our product candidates, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Unless otherwise expressly stated, we obtained this industry, business, market and other data from market research firms and other third parties, including medical publications, government data and similar sources. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information.
You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q completely and with the understanding that our actual future results, performance or achievements may be materially different from what we expect. Except as required by law, we undertake no obligation to update any forward-looking statements to reflect events or circumstances after the date of such statements.
Investors and others should note that we announce material information to our investors using our investor relations website (https://crisprtx.gcs-web.com/), SEC filings, press releases, public conference calls and webcasts. We use these channels as well as social media to communicate with the public about our Company, our business, our product candidates and other matters. It is possible that the information we post on social media could be deemed to be material information. Therefore, we encourage investors, the media, and others interested in our Company to review the information we post on the social media channels listed on our investor relations website.
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Index
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Page Number |
PART I: FINANCIAL INFORMATION |
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Item 1. Condensed Consolidated Financial Statements (unaudited) |
2 |
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Condensed Consolidated Balance Sheets as of March 31, 2026 and December 31, 2025 |
2 |
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Condensed Consolidated Statements of Operations and Comprehensive Loss for the three months ended March 31, 2026 and 2025 |
3 |
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Condensed Consolidated Statements of Shareholders’ Equity for the three months ended March 31, 2026 and 2025 |
4 |
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Condensed Consolidated Statements of Cash Flows for the three months ended March 31, 2026 and 2025 |
5 |
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Notes to Condensed Consolidated Financial Statements |
6 |
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Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations |
19 |
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Item 3. Quantitative and Qualitative Disclosures about Market Risk |
27 |
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Item 4. Controls and Procedures |
27 |
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PART II: OTHER INFORMATION |
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Item 1. Legal Proceedings |
28 |
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Item 1A. Risk Factors |
28 |
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Item 2. Unregistered Sales of Equity Securities and Use of Proceeds |
29 |
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Item 3. Defaults Upon Senior Securities |
29 |
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Item 4. Mine Safety Disclosures |
29 |
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Item 5. Other Information |
29 |
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Item 6. Exhibits |
29 |
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SIGNATURES |
31 |
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PART I—FINANCIAL INFORMATION
Item 1. Financial Statements
CRISPR Therapeutics AG
Condensed Consolidated Balance Sheets
(unaudited, in thousands, except share and per share data)
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As of |
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March 31, |
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December 31, |
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2026 |
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2025 |
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Assets |
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Current assets: |
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Cash and cash equivalents |
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$ |
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$ |
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Marketable securities |
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Prepaid expenses and other current assets |
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Total current assets |
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Property and equipment, net |
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Restricted cash |
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Operating lease assets |
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Other non-current assets |
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Total assets |
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$ |
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$ |
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Liabilities and shareholders’ equity |
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Current liabilities: |
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Accounts payable |
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$ |
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$ |
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Accrued expenses |
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Deferred revenue, current |
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Accrued tax liabilities |
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Operating lease liabilities |
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Other current liabilities |
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Total current liabilities |
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Long-term debt |
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Operating lease liabilities, net of current portion |
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Other non-current liabilities |
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Total liabilities |
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Commitments and contingencies, see Note 7 |
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Shareholders’ equity: |
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Common shares, CHF |
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Treasury shares, at cost, |
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Additional paid-in capital |
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Accumulated deficit |
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Accumulated other comprehensive (loss) income |
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Total shareholders’ equity |
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Total liabilities and shareholders’ equity |
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$ |
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$ |
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The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
2
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CRISPR Therapeutics AG
Condensed Consolidated Statements of Operations and Comprehensive Loss
(unaudited, in thousands, except share and per share data)
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Three Months Ended |
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March 31, |
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2026 |
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2025 |
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Revenue: |
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Collaboration revenue |
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$ |
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$ |
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Grant revenue |
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Total revenue |
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Operating expenses: |
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Research and development |
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General and administrative |
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Collaboration expense, net |
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Total operating expenses |
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Loss from operations |
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( |
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( |
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Other income: |
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Other income, net |
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Total other income, net |
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Net loss before income taxes |
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( |
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( |
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Provision for income taxes |
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( |
) |
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( |
) |
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Net loss |
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( |
) |
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( |
) |
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Foreign currency translation adjustment |
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( |
) |
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Unrealized (loss) gain on marketable securities |
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( |
) |
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Comprehensive loss |
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$ |
( |
) |
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$ |
( |
) |
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Net loss per common share — basic |
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$ |
( |
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$ |
( |
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Basic weighted-average common shares outstanding |
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Net loss per common share — diluted |
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$ |
( |
) |
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$ |
( |
) |
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Diluted weighted-average common shares outstanding |
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The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
3
Table of Contents
CRISPR Therapeutics AG
Condensed Consolidated Statements of Shareholders’ Equity
(unaudited, in thousands, except share and per share data)
|
Common Shares |
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Treasury Shares |
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||||||||||||
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Shares |
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CHF 0.03 |
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Shares |
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Amount, |
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Additional |
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Accumulated |
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Accumulated |
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Total |
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||||||||
Balance at December 31, 2024 |
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$ |
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|
$ |
( |
) |
$ |
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$ |
( |
) |
$ |
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$ |
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||||||
Issuance of common stock, net of issuance costs of $ |
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— |
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— |
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— |
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— |
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Vesting of restricted shares |
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— |
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— |
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— |
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— |
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— |
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Exercise of vested options, net of issuance costs of $ |
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— |
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— |
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— |
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— |
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Purchase of common stock under ESPP |
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— |
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— |
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— |
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— |
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— |
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Stock-based compensation expense |
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— |
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— |
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— |
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— |
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— |
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— |
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Other comprehensive gain |
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— |
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— |
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— |
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— |
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— |
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— |
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Net loss |
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— |
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— |
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— |
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— |
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|
— |
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( |
) |
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— |
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( |
) |
Balance at March 31, 2025 |
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$ |
|
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$ |
( |
) |
$ |
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$ |
( |
) |
$ |
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$ |
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||||||
|
Common Shares |
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Treasury Shares |
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||||||||||||
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Shares |
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CHF 0.03 |
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Shares |
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Amount, |
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Additional |
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Accumulated |
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Accumulated |
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Total |
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||||||||
Balance at December 31, 2025 |
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$ |
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|
|
$ |
( |
) |
$ |
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$ |
( |
) |
$ |
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$ |
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||||||
Vesting of restricted shares |
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— |
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— |
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— |
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— |
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— |
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Exercise of vested options, net of issuance costs of $ |
|
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— |
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— |
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— |
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— |
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Purchase of common stock under ESPP |
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— |
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— |
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— |
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— |
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— |
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Stock-based compensation expense |
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— |
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— |
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— |
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— |
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— |
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— |
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Other comprehensive loss |
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— |
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— |
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— |
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— |
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— |
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— |
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( |
) |
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( |
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Net loss |
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— |
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— |
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— |
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— |
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— |
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( |
) |
|
— |
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( |
) |
Balance at March 31, 2026 |
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$ |
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$ |
( |
) |
$ |
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$ |
( |
) |
$ |
( |
) |
$ |
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|||||
The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
4
Table of Contents
CRISPR Therapeutics AG
Condensed Consolidated Statements of Cash Flows
(unaudited, in thousands)
|
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Three Months Ended March 31, |
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2026 |
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2025 |
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Operating activities: |
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Net loss |
|
$ |
( |
) |
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$ |
( |
) |
Reconciliation of net loss to net cash used in operating activities: |
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Depreciation and amortization |
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Equity-based compensation |
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Other non-cash items, net |
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Changes in: |
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Accounts receivable |
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Prepaid expenses and other assets |
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( |
) |
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( |
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Accounts payable and accrued expenses |
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( |
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Deferred revenue |
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( |
) |
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( |
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Operating lease assets and liabilities |
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( |
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( |
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Other liabilities, net |
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( |
) |
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( |
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Net cash used in operating activities |
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( |
) |
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( |
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Investing activities: |
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Purchase of property, plant and equipment |
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( |
) |
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( |
) |
Investment in equity securities |
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( |
) |
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( |
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Purchases of marketable securities |
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( |
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( |
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Maturities of marketable securities |
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Sales of marketable securities |
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Net cash used in investing activities |
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( |
) |
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( |
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Financing activities: |
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Proceeds from issuance of the Notes |
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Issuance costs and discounts associated with the Notes |
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( |
) |
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(Payments) proceeds from issuance of common shares, net of issuance costs |
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( |
) |
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Proceeds from exercise of options and ESPP contributions, net of issuance costs |
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Net cash provided by financing activities |
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Effect of exchange rate changes on cash |
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( |
) |
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Increase (decrease) in cash |
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( |
) |
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Cash, cash equivalents and restricted cash, beginning of period |
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Cash, cash equivalents and restricted cash, end of period |
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$ |
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$ |
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Supplemental disclosure of non-cash investing and financing activities |
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Equity issuance costs in accounts payable, accrued expenses, and other long-term liabilities |
|
$ |
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$ |
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||
Issuance costs associated with the Notes in accounts payable and accrued expenses |
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$ |
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$ |
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As of March 31, |
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|||||
Reconciliation to amounts within the condensed consolidated balance sheets |
|
2026 |
|
|
2025 |
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||
Cash and cash equivalents |
|
$ |
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$ |
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Prepaid expenses and other current assets |
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Restricted cash |
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Cash, cash equivalents and restricted cash at end of period |
|
$ |
|
|
$ |
|
||
The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
5
Table of Contents
CRISPR Therapeutics AG
Notes to Condensed Consolidated Financial Statements
(unaudited)
1. Basis of Presentation and Significant Accounting Policies
Basis of Presentation
The accompanying condensed consolidated financial statements are unaudited and have been prepared by the Company in accordance with accounting principles generally accepted in the United States of America, or U.S. GAAP.
The accompanying condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The Company views its operations and manages its business in
The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year. These interim financial statements should be read in conjunction with the audited financial statements for the year ended December 31, 2025, which are contained in the 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission, or the SEC, on February 12, 2026.
Use of Estimates
The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, revenue recognition, equity-based compensation expense and reported amounts of expenses during the period. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Changes in estimates are reflected in reported results in the period in which they become known.
Significant Accounting Policies
The significant accounting policies used in preparation of these condensed consolidated financial statements for the three months ended March 31, 2026 are consistent with those discussed in Note 2 to the consolidated financial statements in the Company’s 2025 Annual Report on Form 10-K filed with the SEC on February 12, 2026, with the exception of the new policy below. Any reference in these notes to applicable guidance is meant to refer to the authoritative U.S. GAAP as found in the Accounting Standards Codification, or ASC, and Accounting Standards Updates, or ASUs, of the Financial Accounting Standards Board, or FASB.
Convertible Debt
The Company accounts for its convertible debt instruments as a single unit of accounting, a liability, as the Company concluded that the conversion features do not require bifurcation as a derivative under ASC Topic 815-15, Derivatives and Hedging, as the Company did not issue its convertible debt instruments at a substantial premium. The Company records debt issuance costs as contra-liabilities in its condensed consolidated balance sheets at issuance and amortizes debt issuance costs over the contractual term of the convertible debt instrument using the effective interest rate. The balance of the Company’s Notes (as defined below) presented in its condensed consolidated balance sheets represents the principal balance of the convertible debt instrument less unamortized debt issuance costs. Please refer to Note 8 of the notes to the condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for further information.
Recent Accounting Pronouncements
In November 2024, the FASB issued ASU 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures, and in January 2025, the FASB issued ASU 2025-01, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures: Clarifying the Effective Date. ASU 2024-03 requires disclosure of additional information about specific expense categories in the notes to the financial statements on an interim and annual basis. ASU 2024-03, as clarified by ASU 2025-01, is effective for fiscal years beginning after December 15, 2026, and for interim periods beginning after December 15, 2027, with early adoption permitted. The Company is currently evaluating the disclosure requirements related to this new standard.
2. Marketable Securities
The following table summarizes cash equivalents and marketable securities held at March 31, 2026 and December 31, 2025 (in thousands), which are recorded at fair value. The table below excludes $
6
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and December 31, 2025, respectively.
|
|
Amortized |
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Gross |
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Gross |
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|
Fair Value |
|
||||
March 31, 2026 |
|
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|
|
|
|
|
|
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|
||||
Cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
Corporate debt securities |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Commercial paper |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Government-sponsored enterprise securities |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Total cash equivalents |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Marketable securities: |
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|
|
|
|
|
|
|
|
|
|
|
||||
Corporate debt securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Certificates and term deposits |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Government-sponsored enterprise securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Commercial paper |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total marketable debt securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Corporate equity securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total marketable securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total cash equivalents and marketable securities |
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
|
|||
December 31, 2025 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Money market funds |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
Commercial paper |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total cash equivalents |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Marketable securities: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Corporate debt securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Certificates of deposit |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Government-sponsored enterprise securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Commercial paper |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Total marketable debt securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Corporate equity securities |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Total marketable securities |
|
|
|
|
|
|
|
|
( |
) |
|
|
|
|||
Total cash equivalents and marketable securities |
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
|
|||
The following table summarizes the net unrealized gain (loss) recorded on marketable debt and equity securities during the three months ended March 31, 2026 and 2025 (in millions):
|
|
Three Months Ended |
|
|||||
|
|
March 31, |
|
|||||
|
|
2026 |
|
|
2025 |
|
||
Unrealized (loss) gain recorded on marketable debt securities |
|
$ |
( |
) |
|
$ |
|
|
Unrealized (loss) gain recorded on marketable equity securities |
|
|
( |
) |
|
|
|
|
Unrealized gains and losses on the Company’s marketable debt securities are included in comprehensive loss in the condensed consolidated statements of operations and comprehensive loss. Unrealized gains and losses due to the change in fair value of the Company’s marketable equity securities are included in other income (expense), net, in the condensed consolidated statements of operations and comprehensive loss.
The following table summarizes the net unrealized gain (loss) position of the Company’s marketable debt and equity securities as of March 31, 2026 and December 31, 2025 (in millions):
7
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|
|
March 31, 2026 |
|
|
December 31, 2025 |
|
||
Unrealized (loss) gain position of marketable debt securities |
|
$ |
( |
) |
|
$ |
|
|
Unrealized (loss) gain position of marketable equity securities |
|
|
( |
) |
|
|
|
|
The following table summarizes the aggregate fair value of marketable debt securities that were in an unrealized loss position as of March 31, 2026 and December 31, 2025 by the length of time the security has been in a loss position (in millions):
|
|
March 31, 2026 |
|
|
December 31, 2025 |
|
||
Debt securities in an unrealized loss position for 12 months or less |
|
$ |
|
|
$ |
|
||
Debt securities in an unrealized loss position for more than 12 months |
|
|
|
|
|
|
||
Total debt securities in an unrealized loss position |
|
$ |
|
|
$ |
|
||
The Company determined that there is no material credit risk associated with the above investments as of March 31, 2026. The Company has the intent and ability to hold such securities until recovery. As a result, the Company did not record any charges for credit-related impairments for its marketable securities for the three months ended March 31, 2026 and 2025.
Equity Investments Without Readily Determinable Fair Value
The Company holds investments in privately-held companies in the form of equity securities without readily determinable fair values and in which the Company does not have a controlling interest or significant influence. These investments had a net carrying value of $
3. Fair Value Measurements
The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the fair value hierarchy classification of such fair values as of March 31, 2026 and December 31, 2025 (in thousands):
|
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Fair Value Measurements at |
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|||||||||||||
|
|
March 31, 2026 |
|
|||||||||||||
|
|
Total |
|
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Level 1 |
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Level 2 |
|
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Level 3 |
|
||||
Cash and cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash |
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
— |
|
||
Money market funds |
|
|
|
|
|
|
|
|
— |
|
|
|
— |
|
||
Corporate debt securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Commercial paper |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Government-sponsored enterprise securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Marketable securities: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Corporate debt securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Certificates and term deposits |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Government-sponsored enterprise securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Commercial paper |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Corporate equity securities |
|
|
|
|
|
|
|
|
|
|
|
— |
|
|||
Total |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
— |
|
|||
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
|
|
Fair Value Measurements at |
|
|||||||||||||
|
|
December 31, 2025 |
|
|||||||||||||
|
|
Total |
|
|
Level 1 |
|
|
Level 2 |
|
|
Level 3 |
|
||||
Cash and cash equivalents: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash |
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
— |
|
||
Money market funds |
|
|
|
|
|
|
|
|
— |
|
|
|
— |
|
||
Commercial paper |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Marketable securities: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Corporate debt securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
8
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Certificates of deposit |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Government-sponsored enterprise securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Commercial paper |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Corporate equity securities |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Total |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
Marketable securities classified as Level 1 within the valuation hierarchy consist of corporate equity securities with quoted prices in active markets. Marketable securities classified as Level 2 within the valuation hierarchy generally consist of U.S. treasury securities and government agency securities, certificates of deposit, corporate bonds, commercial paper and warrants to purchase common shares of publicly traded companies. The Company estimates the fair value of these marketable securities by taking into consideration valuations obtained from third-party pricing sources.
Notes
The fair value of the Company’s Convertible Senior Notes due 2031, or the Notes, which differs from their respective carrying values, is determined by prices for the Notes observed in market trading and classified as Level 2 within the valuation hierarchy. The Notes are described in further detail in Note 8 of the notes to the condensed consolidated financial statements included in this Quarterly Report on Form 10-Q.
The following table presents the aggregate face values and the fair values of the Notes, based on their market prices on the last trading day for the periods presented (in thousands):
|
|
March 31, 2026 |
|
|
December 31, 2025 |
|
||||||||||
|
|
Aggregate Face Values |
|
|
Estimated Fair Values |
|
|
Aggregate Face Values |
|
|
Estimated Fair Values |
|
||||
Notes |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
4. Property and Equipment, net
Property and equipment, net, consists of the following (in thousands):
|
|
As of |
|
|||||
|
|
March 31, |
|
|
December 31, |
|
||
|
|
2026 |
|
|
2025 |
|
||
Computer equipment |
|
$ |
|
|
$ |
|
||
Furniture, fixtures and other |
|
|
|
|
|
|
||
Laboratory equipment |
|
|
|
|
|
|
||
Leasehold improvements |
|
|
|
|
|
|
||
Construction work in process |
|
|
|
|
|
|
||
Total property and equipment, gross |
|
|
|
|
|
|
||
Accumulated depreciation |
|
|
( |
) |
|
|
( |
) |
Total property and equipment, net |
|
$ |
|
|
$ |
|
||
Depreciation expense for the three months ended March 31, 2026 and 2025 was $
5. Accrued Expenses
Accrued expenses consist of the following (in thousands):
|
|
As of |
|
|||||
|
|
March 31, |
|
|
December 31, |
|
||
|
|
2026 |
|
|
2025 |
|
||
Payroll and employee-related costs |
|
$ |
|
|
$ |
|
||
Research costs |
|
|
|
|
|
|
||
Collaboration costs |
|
|
|
|
|
|
||
Licensing fees |
|
|
|
|
|
|
||
Professional fees |
|
|
|
|
|
|
||
Intellectual property costs |
|
|
|
|
|
|
||
Other |
|
|
|
|
|
|
||
Total |
|
$ |
|
|
$ |
|
||
9
Table of Contents
6. Significant Contracts
Agreements with Vertex
For purposes of this Note 6 and Note 7, CASGEVY (exagamglogene autotemcel [exa-cel]) is referred to as “CASGEVY”.
2015 collaboration
In 2015, the Company entered into a strategic collaboration, option and license agreement, or the 2015 Collaboration Agreement, with Vertex Pharmaceuticals Incorporated, or Vertex. The 2015 Collaboration Agreement is focused on the use of the Company’s CRISPR/Cas9 gene editing technology to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. The Company and Vertex amended the 2015 Collaboration Agreement in 2017 and 2019 with Amendment No. 1 and Amendment No. 2, respectively, namely to clarify Vertex’s option rights under the 2015 Collaboration Agreement and to modify certain definitions and provisions of the 2015 Collaboration Agreement to make them consistent with the JDA (as defined below) and a strategic collaboration and license agreement from 2019 for the development and commercialization of products for the treatment of Duchenne muscular dystrophy, or DMD, and myotonic dystrophy type 1, or DM1. In 2017, Vertex exercised an option granted to it under the 2015 Collaboration Agreement to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, and in 2019, Vertex exercised the remaining options granted to it under the 2015 Collaboration Agreement to exclusively license certain collaboration targets developed under the 2015 Collaboration Agreement.
Hemoglobinopathies collaboration
In 2017, following Vertex’s exercise of its option to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, the Company and Vertex entered into a joint development and commercialization agreement, or the JDA, and agreed for potential hemoglobinopathy treatments, including CASGEVY, the Company and Vertex would share equally all research and development costs and worldwide revenues. In 2021, the Company and Vertex amended and restated the JDA, as amended and in effect, from time to time, or the A&R Vertex JDCA, pursuant to which the parties agreed to, among other things, (a) adjust the governance structure for the collaboration and adjust the responsibilities of each party thereunder, whereby Vertex leads and has all decision making (i.e., control) in relation to the CASGEVY program prospectively; (b) adjust the allocation of net profits and net losses between the parties with respect to CASGEVY only, which will be allocated
In December 2023, the Company entered into an amendment to the A&R Vertex JDCA, or Amendment No. 1 to the A&R Vertex JDCA, with Vertex related to the global development, manufacturing, and commercialization of CASGEVY. Pursuant to Amendment No. 1 to the A&R Vertex JDCA, among other things, the Company and Vertex agreed to (a) allocate certain costs arising from a license agreement with a third party, resulting in a current payment due to Vertex by the Company of $
Letter Agreement
In May 2024, Vertex and the Company entered into a letter agreement, or the Letter Agreement, with respect to the priority review voucher issued by the U.S. Food and Drug Administration to Vertex as the sponsor of the rare pediatric disease product application for CASGEVY. Vertex and the Company agreed that if Vertex utilizes or transfers the priority review voucher prior to the first calendar year in which the CASGEVY program generates a net profit, Vertex will pay the Company $
Collaboration in the field of diabetes
In 2021, the Company and ViaCyte, Inc., or ViaCyte, entered into a joint development and commercialization agreement, as amended and in effect, from time to time, or the ViaCyte JDCA, to jointly develop and commercialize product candidates and shared products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. In the third quarter of 2022, Vertex acquired ViaCyte, and ViaCyte became a wholly-owned subsidiary of Vertex. In March 2023, (1) the Company and ViaCyte entered into an amendment to the ViaCyte JDCA, or the ViaCyte JDCA Amendment, and adjusted certain rights and obligations of the Company and ViaCyte under the ViaCyte JDCA, and (2) the Company and Vertex entered into a non-exclusive license agreement, or the Non-Ex License Agreement, pursuant to which the Company agreed to license to Vertex, on a non-exclusive basis, certain of its gene editing intellectual property to exploit certain products for the
10
Table of Contents
diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. Subsequently, ViaCyte elected to opt-out of the ViaCyte JDCA. Per the opt-out terms, the on-going collaboration assets are now wholly owned by the Company, subject to a royalty on future sales owed to ViaCyte. The opt-out became effective in early February 2024.
Accounting Analysis
For purposes of this Note 6, the 2015 Collaboration Agreement, Amendment No. 1, Amendment No. 2, A&R Vertex JDCA, and Amendment No. 1 to the A&R Vertex JDCA are collectively referred to as the “Vertex Hemoglobinopathy Agreements” and the Non-Ex License Agreement and ViaCyte JDCA Amendment are collectively referred to as the “March 2023 Diabetes Agreements.”
The Vertex Hemoglobinopathy Agreements and the March 2023 Diabetes Agreements include components of a customer-vendor relationship as defined under ASC 606, Revenue from Contracts with Customers, or ASC 606, collaborative arrangements as defined under ASC 808, Collaborative Agreements, or ASC 808, and research and development costs as defined under ASC 730, Research and Development, or ASC 730. Specifically, with regards to the March 2023 Diabetes Agreements, the Company concluded that the non-exclusive license is a performance obligation under ASC 606 and the ongoing research and development services under the ViaCyte JDCA Amendment are a unit of account under ASC 808.
The Company has determined that recognition criteria for the Letter Agreement has not been met and will not be met until the priority review voucher is (i) utilized or (ii) there is sufficient profitability such that Vertex is obligated to pay the Company under the Letter Agreement.
Accounting Analysis Under ASC 606
Accounting for the Vertex Hemoglobinopathy Agreements
Recognition of Revenue
Milestones under the Vertex Hemoglobinopathy Agreements
The Company has evaluated the milestones that may be received in connection with the Vertex Hemoglobinopathy Agreements.
Under the 2015 Collaboration Agreement and subsequent amendments, the Company is eligible to receive up to $
The Company has the option to conduct research at its own cost in certain defined areas. If such research is beneficial to the CASGEVY program and CASGEVY ultimately achieves regulatory approval in such areas, the Company could be entitled to receive from Vertex certain milestone payments aggregating to high eight digits.
Each of the remaining milestones described above are fully constrained as of March 31, 2026. There is uncertainty as to whether the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.
Accounting Analysis under ASC 808
Vertex Hemoglobinopathy Agreements
In connection with the Vertex Hemoglobinopathy Agreements, the Company identified the following collaborative elements, which are accounted for under ASC 808: (i) development and commercialization services for shared products, including any transition services related to CASGEVY under the A&R Vertex JDCA; (ii) R&D Services for follow-on products; and (iii) committee participation. The related impact of the cost sharing under the Vertex Hemoglobinopathy Agreements is included within collaboration expense, net, in the condensed consolidated statements of operations and comprehensive loss.
During the three months ended March 31, 2026 and 2025, the Company recognized $
11
Table of Contents
Additional Accounting Considerations
The Company is eligible to receive potential future payments of up to $
Each of the remaining milestones are fully constrained as of March 31, 2026. There is uncertainty that the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.
As of March 31, 2026, there was $
As of March 31, 2026, the Company is eligible to receive potential future milestone payments from Vertex of up to $
Each of the remaining milestones under the Non-Ex License Agreement are fully constrained as of March 31, 2026. There is uncertainty as to whether the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.
Agreement with Sirius Therapeutics
In May 2025, the Company entered into a collaboration, option and license agreement, or the Sirius Agreement, with Sirius Therapeutics and certain of its affiliates, or Sirius, pursuant to which, among other things, (1) Sirius and the Company will collaborate on the research, development, manufacture, commercialization and use of certain collaboration products utilizing Sirius’ siRNA technology for targeting Factor XI, including CTX611, collectively, the Sirius Collaboration Products; and (2) Sirius granted to the Company options to exclusively license Sirius siRNA technology to target up to two licensed targets from a list of seven reserved targets for the research, development, manufacture and commercialization of licensed products, collectively the siRNA Licensed Products, in exchange for the potential to receive certain option fees, milestone payments and royalties.
In connection with entering into the Sirius Agreement, the Company made an upfront cash payment to Sirius of $
With respect to Sirius Collaboration Products, the Company and Sirius will share equally all development and commercialization costs. For the first Sirius Collaboration Product successfully developed, the Company will be the lead party responsible for Phase 3 global development and commercialization efforts in the United States and Sirius will be the lead party responsible for Phase 3 development (subject to the global development plan) and commercialization efforts in Greater China. The parties will determine the lead party responsible for commercialization in the rest of the world at a future date. The Company and Sirius will share equally net profits and net losses incurred under the Sirius Agreement with respect to all Sirius Collaboration Products, except in the event that a party opts out of the joint development and commercialization. The Company will pay Sirius certain specified future development and regulatory milestones of up to an aggregate of $
With respect to the siRNA Licensed Products, if the Company elects to exercise its option to a licensed target to research, develop, manufacture and commercialize siRNA Licensed Products, the Company will make a one-time $
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royalty payments in the mid-single digits to low double digits range on future sales of a commercialized siRNA Licensed Product. The royalty payments are subject to reduction under certain specified conditions set forth in the Sirius Agreement. In addition, at the Company’s sole election, such milestones may be paid in cash, common shares of the Company, or a combination thereof. The Company is solely responsible for all research, development, manufacturing and global commercialization activities and associated costs for the siRNA Licensed Products, as well as all associated costs related to Sirius activities set forth in any applicable research plan relating thereto.
Accounting for the Sirius Agreement
The Sirius Agreement includes components of collaborative arrangements as defined under ASC 808 and research and development costs as defined under ASC 730.
Specifically, development and commercialization costs for the Sirius Collaboration Products contain collaborative elements accounted for under ASC 808, and the related impact of cost sharing for the Sirius Collaboration Products under the Sirius Agreement is included within research and development expenses in the condensed consolidated statements of operations and comprehensive loss. Costs related to siRNA Licensed Products under the Sirius Agreement are included within research and development expenses in the consolidated statement of operations and comprehensive loss.
Research and development costs under the Sirius Agreement were $
7. Commitments and Contingencies
Leases
Refer to Note 7 to the consolidated financial statements in the Company’s 2025 Annual Report on Form 10-K filed with the SEC on February 12, 2026 for discussion of the Company’s lease arrangements.
Litigation
In the ordinary course of business, the Company is or has been from time to time involved in lawsuits, investigations, proceedings and threats of litigation related to, among other things, the Company’s intellectual property estate (including certain in-licensed intellectual property), commercial arrangements and other matters. Such proceedings may include quasi-litigation, inter partes administrative proceedings in the U.S. Patent and Trademark Office and the European Patent Office or patent offices in other countries, involving the Company’s intellectual property estate including certain in-licensed intellectual property. For example, in the fourth quarter of 2025, ToolGen, Inc., or ToolGen, initiated a lawsuit against the Company and other third parties alleging patent infringement by CASGEVY of a ToolGen patent relating to CRISPR/Cas9 gene editing technology. The Company was subsequently dismissed from the lawsuit as a defendant in April 2026 without prejudice. The outcome of any of the foregoing is inherently uncertain. In addition, litigation and related matters are costly and may divert the attention of Company’s management and other resources that would otherwise be engaged in other activities. If the Company is unable to prevail in any such proceedings, the Company’s business, results of operations, liquidity and financial condition could be adversely affected.
Letters of Credit
The Company had restricted cash of $
Research, Manufacturing, License and Intellectual Property Agreements
The Company has engaged several research institutions and companies to identify new delivery strategies and applications of the Company’s gene editing technology. The Company is also a party to a number of license agreements which require significant upfront payments and may be required to make future royalty payments and potential milestone payments from time to time. In addition, the Company is also a party to intellectual property agreements, which require maintenance and milestone payments from time to time. Further, the Company is a party to a number of manufacturing agreements that require upfront payments for the future performance of services.
In connection with these agreements, on a product-by-product basis, the counterparties are eligible to receive up to low eight-digit potential payments upon specified research, development and regulatory milestones. In addition, on a product-by-product basis, the counterparties are eligible to receive potential commercial milestone payments based on specified annual sales thresholds. The potential payments are low-single digit percentages of the specified annual sales thresholds. The counterparties are also eligible to receive low single-digit royalties on future net sales.
In 2025, a third-party licensor formally engaged with the Company regarding certain matters under their intellectual property
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contracts with the Company that may lead to further actions that could result in additional amounts being owed by the Company to such third party. The Company determined that it was probable that a loss was incurred as of December 31, 2025 and, based on the Company’s best estimate of the loss, the Company recorded incremental research and development expenses of $
Under certain circumstances and if certain contingent future events occur, Vertex is eligible to receive up to $
Under the A&R Vertex JDCA for 2022, 2023 and 2024, the Company had an option to defer a portion of its share of costs if spending on the CASGEVY program exceeded specified amounts, which the Company exercised in each such year, resulting in deferred costs of $
The Company may be required to make future potential payments to Sirius under the Sirius Agreement defined and described in Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q. Potential payments to Sirius include (i) up to $
8. Debt
Notes, net
In March 2026, the Company issued $
The Notes are senior, unsecured obligations of the Company and will mature on March 1, 2031, or the maturity date, unless earlier converted, redeemed or repurchased. The Notes will accrue interest payable semiannually in arrears on March 1 and September 1 of each year, beginning on September 1, 2026. Holders may convert all or any portion of their Notes at their option prior to the maturity date, other than during a “conversion freeze period” (as defined in the Indenture). Upon conversion, the Company will deliver common shares at an initial conversion rate of
In addition, if certain corporate events occur or are anticipated to occur prior to the maturity date or if the Company delivers a notice of optional redemption, the Company will, in certain circumstances, increase the conversion rate for a holder who elects to convert its Notes in connection with such a corporate event or called (or deemed called) for redemption in connection with such notice of optional redemption. Initially, a maximum of
The Company may not redeem the Notes prior to
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(whether or not consecutive) during any
If the Company undergoes a fundamental change (as defined in the Indenture), then, subject to certain conditions and except as described in the Indenture, holders may require the Company to repurchase for cash all or any portion of their Notes at a fundamental change repurchase price equal to
The Indenture includes customary covenants and sets forth certain events of default after which the Notes may be declared immediately due and payable and sets forth certain types of bankruptcy or insolvency events of default involving the Company after which the Notes become automatically due and payable.
The Company received net proceeds from the 2026 Note Offering of approximately $
Additional Information Related to the Notes
The outstanding balances of the Notes consisted of the following (in thousands):
|
|
March 31, 2026 |
|
|
December 31, 2025 |
|
||
Principal |
|
$ |
|
|
$ |
|
||
Unamortized debt discount and issuance costs |
|
|
( |
) |
|
|
|
|
Net carrying amount |
|
$ |
|
|
$ |
|
||
The following table sets forth the total interest expense recognized and effective interest rates related to the Notes for the periods presented (in thousands):
|
|
Three Months Ended March 31 |
|
|||||
|
|
2026 |
|
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2025 |
|
||
Contractual interest expense |
|
$ |
|
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$ |
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||
Amortization of debt discount and issuance costs |
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|
|
|
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||
Total interest and amortization expense |
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$ |
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$ |
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||
Effective interest rate |
|
|
% |
|
|
|
||
As of March 31, 2026, interest payable on the Notes was $
9. Share Capital
All of the Company’s common shares are authorized under Swiss corporate law with a nominal value of
As of March 31, 2026, the Company’s share capital consists of
Common Share Issuances
At-the-Market Offerings (ATM)
The Company has entered into an Open Market Sale AgreementSM, or the Sales Agreement, with Jefferies LLC under which the Company, at its sole discretion, is able to offer and sell, from time to time at prevailing market prices, its common shares. The following are in connection with the Sales Agreement.
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2021 ATM
In January 2021, the Company filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $
For the three months ended March 31, 2025, the Company issued and sold an aggregate of
As of December 31, 2025, the Company had issued and sold an aggregate of
2025 ATM
In October 2025, the Company filed a new prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $
Share Issuance Agreement with Sirius Therapeutics
As described in Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q, the Company and Sirius entered into a share issuance agreement in 2025 and the Company registered and issued
10. Stock-based Compensation
During the three months ended March 31, 2026 and 2025, the Company recognized the following stock-based compensation expense (in thousands):
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Three Months Ended March 31, |
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|||||
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2026 |
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2025 |
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||
Research and development |
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$ |
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$ |
|
||
General and administrative |
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Total |
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$ |
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$ |
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||
Stock option activity
The following table summarizes stock option activity for the three months ended March 31, 2026:
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Shares |
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Weighted- |
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||
Outstanding at December 31, 2025 |
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$ |
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||
Granted |
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Exercised |
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( |
) |
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Cancelled or forfeited |
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( |
) |
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Outstanding at March 31, 2026 |
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$ |
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||
Exercisable at March 31, 2026 |
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$ |
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Vested and expected to vest at March 31, 2026 |
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$ |
|
||
As of March 31, 2026, total unrecognized compensation expense related to stock options was $
Restricted stock activity
The following table summarizes restricted stock activity for the three months ended March 31, 2026:
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Shares |
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Weighted- |
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||
Unvested balance at December 31, 2025 |
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$ |
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Granted |
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Vested |
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( |
) |
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Cancelled or forfeited |
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( |
) |
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Unvested balance at March 31, 2026 |
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|
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$ |
|
||
As of March 31, 2026, total unrecognized compensation expense related to unvested restricted common shares was $
11. Net Loss Per Share Attributable to Common Shareholders
Basic net loss per share is calculated by dividing net loss attributable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net loss per share is calculated by adjusting net loss to remove the effects from potential dilutive common shares and dividing this adjusted amount by the weighted average number of common shares and potential dilutive common shares outstanding for the period. For purposes of the diluted net loss per share calculation, stock options, unvested restricted common shares, ESPP shares and shares issuable under an assumed conversion of the Notes are considered to be common stock equivalents but are excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive; therefore, basic and diluted net loss per share were the same for all periods presented as a result of the Company’s net loss. The Company’s net loss is net loss attributable to common shareholders for all periods presented.
The Company did not include the securities in the following table in the computation of the net loss per share calculations for the three months ended March 31, 2026 and 2025 because the effect would have been anti-dilutive during each period:
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March 31, |
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|||||
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2026 |
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2025 |
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Outstanding options |
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Unvested restricted common shares |
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ESPP |
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Assumed conversion of the Notes |
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Total |
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12. Income Taxes
During the three months ended March 31, 2026 and 2025, the Company recorded an income tax provision of $
13. Segment Information
The Company operates as
The measure of segment assets is reported on the condensed consolidated balance sheets as total consolidated assets. All material long-lived assets are located in the United States. Long-lived assets consist of property and equipment, net, and operating lease right-of-use assets.
The following table illustrates information about segment revenue, significant segment expenses and segment operating loss for the periods presented (in thousands):
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|
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Three Months Ended March 31, |
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2026 |
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2025 |
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Revenue: |
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Collaboration revenue |
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$ |
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$ |
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Grant revenue |
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Less1: |
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Research and development expense2 |
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General and administrative expense3 |
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Collaboration expense, net - Vertex4 |
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Collaboration expense, net - Sirius5 |
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Stock-based compensation expense |
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Depreciation expense |
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Other segment items6 |
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( |
) |
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( |
) |
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Segment net loss |
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( |
) |
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( |
) |
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Reconciliation of profit or loss: |
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Adjustments or reconciling items |
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Consolidated net loss |
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( |
) |
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( |
) |
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(1) The significant expense categories and amounts align with the segment-level information that is regularly provided to the CODM.
(2) Research and development expense for the three months ended March 31, 2026 and 2025 excludes $
(3) General and administrative expense for the three months ended March 31, 2026 and 2025 excludes $
(4) Collaboration costs specific to the Vertex Hemoglobinopathy Agreements (as defined in Note 6) accounted for under ASC 808 are presented within “collaboration expense, net” in the condensed consolidated statements of operations and comprehensive loss.
(5) In the second quarter of 2025, the Company entered into the Sirius Agreement (as defined in Note 6). Collaboration costs under the Sirius Agreement are presented within “research and development expense” in the condensed consolidated statements of operations and comprehensive loss. In the first quarter of 2026, the significant expense categories reviewed by the CODM were updated to include collaboration costs associated with the Sirius Agreement.
(6) Other segment items include interest income (expense), net, the change in fair value of corporate equity securities and income tax expense.
The Company operates in the United States and Switzerland. Collaboration revenue is attributed to the CRISPR Therapeutics AG entity, which is domiciled in Switzerland.
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Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with (i) our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and (ii) our audited consolidated financial statements and related notes and management’s discussion and analysis of financial condition and results of operations included in our Annual Report on Form 10-K for the year ended December 31, 2025 filed with the Securities and Exchange Commission, or the SEC, on February 12, 2026. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business and impact and potential impacts on our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including, without limitation, those factors set forth in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2025 and the “Risk Factors” section of subsequent Quarterly Reports on Form 10-Q, our actual results or timing of certain events could differ materially from the results or timing described in, or implied by, these forward-looking statements.
Overview
Our mission is to create transformative gene-based medicines for serious human diseases. We are a leading biopharmaceutical company focused on the development of CRISPR-based therapeutics, including by using CRISPR/Cas9 technology. We have established a portfolio of therapeutic programs spanning four core franchises: hemoglobinopathies, in vivo approaches, CAR-T and regenerative medicine. Depending on the program, we take either an ex vivo approach, in which we edit cells outside of the human body before administering them to the patient, or an in vivo editing approach, where we deliver the CRISPR-based therapeutic directly to target cells within the human body.
CRISPR/Cas9 is a revolutionary technology for gene editing, the process of precisely altering specific sequences of genomic DNA. We have advanced this technology from discovery to an approved medicine with unparalleled speed, culminating in the landmark first approval of a CRISPR-based therapy, CASGEVY (exagamglogene autotemcel [exa-cel]), in 2023 with our collaborators at Vertex Pharmaceuticals Incorporated, or Vertex.
We continue to innovate on our platform to develop next-generation technologies that can enable new therapies. We are developing other technologies, including delivery technologies and other gene editing technologies, like SyNTase. Through our efforts, we aim to unlock the full potential of gene-based therapeutics to create medicines that can transform people’s lives. We believe that our innovative research, translational expertise and clinical development experience position us as a leader in the development of CRISPR-based therapeutics and may enable us to create an entirely new class of highly effective and potentially curative therapies for patients with both common and rare diseases for whom current biopharmaceutical approaches have had limited success.
Hemoglobinopathies
CASGEVY
CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin in red blood cells, which can compensate for the defective adult hemoglobin in patients with sickle cell disease, or SCD, or transfusion-dependent beta thalassemia, or TDT. CASGEVY is the first therapy to emerge from our strategic partnership with Vertex and is being advanced under a joint development and commercialization agreement between us and Vertex and certain of its affiliates.
In 2023, CASGEVY became the first-ever approved CRISPR-based gene-editing therapy in the world. To date, CASGEVY has been approved in the United States, European Union, Great Britain, Canada, Switzerland and certain countries in the Middle East for the treatment of eligible patients 12 years and older with SCD or TDT. We and Vertex continue to investigate CASGEVY, including in clinical trials designed to assess the safety and efficacy of a single dose of CASGEVY in patients 12 to 35 years of age with severe SCD and TDT, respectively, two pivotal trials in patients 5 to 11 years of age, one in severe SCD and a second in TDT, and long-term follow-up clinical trials designed to follow participants for up to 15 years after CASGEVY infusion. Overall, CASGEVY safety data presented to date is generally consistent with an autologous stem cell transplant and myeloablative conditioning. Efficacy data presented to date support the profile of this therapy as a potential one-time functional cure for people with severe SCD and TDT.
Additional candidates
We continue to advance our internally developed targeted conditioning program, as well as in vivo hematopoietic stem cell editing approaches utilizing lipid nanoparticle-mediated delivery through preclinical studies. Both initiatives could significantly expand the addressable patient populations for SCD and TDT.
In Vivo Liver Editing
We have established a leading platform for in vivo gene editing and are rapidly advancing a pipeline of in vivo gene editing candidates that target the liver, taking advantage of validated lipid nanoparticle, or LNP, delivery technologies, and aim to treat
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diseases where we can produce a strong therapeutic effect by safely disrupting a gene with well-understood genetic association. We have established a proprietary LNP delivery platform to enable gene-editing in the liver using both CRISPR/Cas9 and our novel, proprietary SyNTase editing technologies.
Our in vivo portfolio includes cardiovascular programs, such as CTX310, directed towards angiopoietin-related protein 3, which is currently in an ongoing Phase 1b clinical trial in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia.
Additional candidates
In addition, we have a number of earlier stage investigational in vivo programs leveraging gene disruption in the liver for both common and rare diseases, including CTX340, directed towards angiotensinogen for the treatment of refractory hypertension; our next-generation LPA program, CTX321 directed towards LPA, the gene encoding apolipoprotein(a), a major component of lipoprotein(a), or Lp(a), in development for patients with elevated Lp(a), and CTX460, directed towards SERPINA1 using our proprietary SyNTase editing platform, for the treatment of alpha-1 antitrypsin deficiency. CTX340, CTX321 and CTX460 are currently in IND/CTA-enabling studies. We are also pursuing additional delivery technologies, including LNPs, for delivery to tissues beyond the liver, including hematopoietic stem cells and T cells.
siRNA-based Programs
Our siRNA-based portfolio includes clinical-stage programs in cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics and certain of its affiliates, or Sirius.
CTX611 is a novel double-stranded, long-acting siRNA, designed to target the human coagulation factor XI, or FXI, messenger RNA and inhibit FXI protein expression. Through modulation of the intrinsic coagulation pathway, CTX611 is intended to provide anticoagulant and antithrombotic effects. Supported by clinical experience conducted by Sirius in two Phase 1 clinical trials, CTX611 is being developed as a long-acting FXI inhibitor with the potential to support infrequent, including semi-annual, subcutaneous administration.
CTX611 is in ongoing Phase 2 clinical trials, including in patients undergoing total knee arthroplasty.
CAR-T
We believe CRISPR/Cas9 has the potential to create the next generation of CAR-T cell therapies that may have a superior product profile and allow broader patient access compared to current autologous therapies. We are advancing several cell therapy programs in both autoimmune and immuno-oncology indications.
Zugocabtagene geleucel
Our lead next-generation product candidate, zugocabtagene geleucel (zugo-cel; formerly CTX112), incorporates edits designed to enhance CAR-T potency, reduce CAR-T exhaustion and evade the immune system. As a result of the next-generation edits, zugo-cel exhibits increased manufacturing robustness, with a higher and more consistent number of CAR-T cells produced per batch. We are producing zugo-cel for clinical trials at our internal GMP manufacturing facility in Framingham, Massachusetts. Zugo-cel continues to advance in both autoimmune disease and hematologic malignancies.
In autoimmune disease, it is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the product candidate in adult patients across multiple indications, including an initial clinical trial in systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis; a second clinical trial in immune thrombocytopenia purpura and warm autoimmune hemolytic anemia; and a third clinical trial in autoimmune neurologic diseases, including progressive multiple sclerosis, neuromyelitis optica spectrum disorder, myelin oligodendrocyte glycoprotein antibody-associated Disease (MOGAD), N-methyl-D-aspartate receptor (NMDAR) and leucine-rich glioma-inactivated Protein 1 (LGI1) autoimmune encephalitis, and stiff person syndrome.
In immuno-oncology, the Phase 1/2 clinical trial in adult patients with relapsed or refractory B-cell malignancies who have received at least two prior lines of therapy is ongoing. Eligible disease subtypes include large B-cell lymphoma, or LBCL, follicular lymphoma grade 1-3a, marginal zone lymphoma, and mantle cell lymphoma. Initial positive clinical data generated through December 2025 support the advancement of zugo-cel into the Phase 2 portion of the ongoing Phase 1/2 trial. We have also established a collaboration and clinical supply agreement with Eli Lilly to evaluate zugo-cel together with pirtobrutinib in aggressive B-cell lymphomas, further expanding the program’s development in oncology. Zugo-cel has been granted regenerative medicine advanced therapy designation by the U.S. Food and Drug Administration for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma.
Additional candidates
Our CRISPR/Cas9 platform enables us to innovate continuously by incorporating incremental edits into next-generation products. We are advancing several additional investigational CAR-T programs. In addition, we are developing both transient and integrated in vivo CAR-T therapies by targeting T cells with LNPs and leveraging our delivery, mRNA, and gene editing expertise.
Regenerative Medicine
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We continue to advance our regenerative medicine portfolio, including in diabetes. We are advancing CTX213, a deviceless beta cell replacement product candidate consisting of unencapsulated precursor islet cells derived from induced pluripotent stem cells for the treatment of type 1 diabetes, or T1D. To date, CTX213 has demonstrated preclinical efficacy data via direct administration. In addition, we have granted a non-exclusive license to certain of our CRISPR/Cas9 intellectual property to Vertex to accelerate Vertex’s development of hypoimmune cell therapies for T1D in exchange for certain milestones and royalties.
Next-generation Editing Modalities
While we have made significant progress with our current portfolio of programs, we recognize that we may be able to bring transformative therapies to even more patients by continuing to innovate to unlock the full potential of gene editing. We are focused on innovating next-generation editing modalities. For example, we have developed a proprietary, next-generation, site-specific gene correction platform called SyNTase editing. In addition, we are also developing technologies to enable whole gene correction and insertion via non-viral DNA delivery and all-RNA systems.
Partnerships
Given the numerous potential therapeutic applications for CRISPR/Cas9, we have partnered strategically to broaden the indications we can pursue and accelerate development of programs by accessing specific technologies and/or disease-area expertise. We maintain broad partnerships to develop gene-based therapeutics in specific disease areas.
Hemoglobinopathies. In 2015, we partnered with Vertex and entered into a strategic collaboration, option and license agreement, which focused on the discovery and development of gene-based treatments for hemoglobinopathies and cystic fibrosis using CRISPR/Cas9 gene-editing technology. In 2017, Vertex exercised its option to co-develop and co-commercialize the hemoglobinopathies program and we entered into a joint development and commercialization agreement with Vertex, which we amended and restated in 2021, pursuant to which, among other things, we are co-developing and co-commercializing CASGEVY for TDT and SCD.
siRNA. In May 2025, we partnered with Sirius and entered into the Sirius Agreement pursuant to which, among other things, (1) we and Sirius will collaborate on the research, development, manufacture, commercialization and use of the Sirius Collaboration Products, including co-development and co-commercialization of CTX611; and (2) Sirius granted us options to exclusively license Sirius siRNA technology to target up to two licensed targets from a list of seven reserved targets for the research, development, manufacture and commercialization of siRNA Licensed Products. For the first Sirius Collaboration Product successfully developed, we will be the lead party responsible for Phase 3 global development and commercialization efforts in the United States and Sirius will be the lead party responsible for Phase 3 development (subject to the global development plan) and commercialization efforts in Greater China.
Other Partnerships. We have entered into a number of additional collaborations, research and license agreements in other therapeutic areas, including additional agreements with Vertex including for the treatment of Duchenne muscular dystrophy and myotonic dystrophy type 1, as well as diabetes, and others, including to support and complement our hematopoietic stem cell, CAR-T, in vivo and diabetes programs and platform.
Financial Overview
Since our inception in October 2013, we have devoted substantially all of our resources to our research and development efforts, identifying potential product candidates, undertaking drug discovery and preclinical development activities, building and protecting our intellectual property estate, establishing internal manufacturing capabilities, organizing and staffing our company, business development activities, business planning, raising capital and providing general and administrative support for these operations. To date, we have primarily financed our operations through private placements of our preferred shares, common share issuances, convertible loans, issuance of convertible notes and payments related to certain of our license and collaboration agreements with strategic partners.
We have a history of recurring losses and expect to continue to incur losses for the foreseeable future; however, we have been in a net income position in certain previous years due to certain payments associated with our collaboration and license agreements with Vertex. Our net losses may fluctuate significantly from quarter to quarter and year to year. We anticipate that our expenses will increase as we continue our current research programs and development activities; seek to identify additional research programs and additional product candidates; conduct initial drug application supporting preclinical studies and initiate clinical trials for our product candidates; pursue business development activities; initiate preclinical testing and clinical trials for any other product candidates we identify and develop; seek regulatory approval for our product candidates; maintain, defend, protect and expand our intellectual property estate; further develop our gene editing platform and other technologies; hire additional research, clinical and scientific personnel; incur facilities costs associated with such personnel growth; continue to develop internal manufacturing capabilities and infrastructure; and incur additional costs associated with operating as a public company.
Revenue Recognition
We have not generated any revenue to date from sales of any wholly-owned product and do not expect to do so in the near
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future. Revenue recognized for the three months ended March 31, 2026 and 2025 was not material. For additional information about our revenue recognition policy, see Note 2, “Summary of Significant Accounting Policies,” in our Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on February 12, 2026, as well as Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q.
Research and Development Expenses
Research and development expenses consist primarily of costs incurred for our research activities, including our product discovery efforts and the development of our product candidates, which include:
Our external research and development expenses support our various preclinical and clinical programs, and, as such, we do not break down external research and development expenses further. Our internal research and development expenses consist of payroll and benefits expenses, facilities expense, and other indirect research and development expenses incurred in support of overall research and development activities and, as such, are not allocated to a specific development stage or therapeutic area. Research and development costs are expensed as incurred. Nonrefundable advance payments for research and development goods or services to be received in the future are deferred and capitalized. The capitalized amounts are expensed as the related goods are delivered or the services are performed. At this time, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of any product candidates we may identify and develop. This is due to the numerous risks and uncertainties associated with developing such product candidates, including the uncertainty of:
A change in the outcome of any of these variables with respect to the development of any product candidates or the subsequent commercialization of any product candidates we may successfully develop could significantly change the costs, timing and viability associated with the development of that product candidate.
Research and development activities are central to our business model. We expect to continue to incur research and development costs consistent with research and development at companies of our size and stage of development, which may increase in the foreseeable future as our current development programs progress, new programs are added and we continue to prepare regulatory filings. These increases will likely include the costs related to the implementation and expansion of clinical trial sites and related patient enrollment, monitoring, program management and manufacturing expenses for current and future clinical trials.
General and Administrative Expenses
General and administrative expenses consist primarily of employee-related expenses, including salaries, benefits and equity-based compensation, for personnel in executive, finance, accounting, business development, human resources and other general and administrative functions. Other significant costs include facility costs not otherwise included in research and development expenses, legal fees relating to patent and corporate matters and fees for accounting and consulting services.
We expect to continue to incur general and administrative expenses consistent with general and administrative functions at research and development companies of our size and stage of development, which may increase in the future to support continued
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research and development activities, and potential commercialization of our product candidates. In addition, we anticipate ongoing expenses related to the reimbursements of third-party patent related expenses in connection with certain of our in-licensed intellectual property.
Collaboration Expense, Net
Collaboration expense, net, consists of operating expense under our collaboration with Vertex for the hemoglobinopathies program.
Other Income (Expense), Net
Other income (expense), net consists primarily of interest expense on debt, interest income earned on investments, as well as the change in fair value of corporate equity securities.
Results of Operations
Comparison of three months ended March 31, 2026 and 2025 (in thousands):
|
|
Three Months Ended March 31, |
|
|
Period to Period |
|
||||||
|
|
2026 |
|
|
2025 |
|
|
Change |
|
|||
Revenue: |
|
|
|
|
|
|
|
|
|
|||
Collaboration revenue |
|
$ |
1,000 |
|
|
$ |
— |
|
|
$ |
1,000 |
|
Grant revenue |
|
|
458 |
|
|
|
865 |
|
|
|
(407 |
) |
Total revenue |
|
|
1,458 |
|
|
|
865 |
|
|
|
593 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|||
Research and development |
|
|
68,574 |
|
|
|
72,484 |
|
|
|
(3,910 |
) |
General and administrative |
|
|
17,183 |
|
|
|
19,296 |
|
|
|
(2,113 |
) |
Collaboration expense, net |
|
|
45,949 |
|
|
|
57,509 |
|
|
|
(11,560 |
) |
Total operating expenses |
|
|
131,706 |
|
|
|
149,289 |
|
|
|
(17,583 |
) |
Loss from operations |
|
|
(130,248 |
) |
|
|
(148,424 |
) |
|
|
18,176 |
|
Other income, net |
|
|
8,156 |
|
|
|
13,537 |
|
|
|
(5,381 |
) |
Loss before income taxes |
|
|
(122,092 |
) |
|
|
(134,887 |
) |
|
|
12,795 |
|
Provision for income taxes |
|
|
(839 |
) |
|
|
(1,109 |
) |
|
|
270 |
|
Net loss |
|
$ |
(122,931 |
) |
|
$ |
(135,996 |
) |
|
$ |
13,065 |
|
Collaboration Revenue
Collaboration revenue was not material for the three months ended March 31, 2026 and 2025.
Research and Development Expenses
Research and development expenses were $68.6 million for the three months ended March 31, 2026, compared to $72.5 million for the three months ended March 31, 2025. The following table summarizes our research and development expenses for the three months ended March 31, 2026 and 2025, together with the changes in those items in dollars (in thousands):
|
|
Three Months Ended March 31, |
|
|
Period to Period |
|
||||||
|
|
2026 |
|
|
2025 |
|
|
Change |
|
|||
External research and development expenses |
|
$ |
21,450 |
|
|
$ |
17,852 |
|
|
$ |
3,598 |
|
Employee-related expenses |
|
|
14,991 |
|
|
|
19,735 |
|
|
|
(4,744 |
) |
Facility expenses |
|
|
22,234 |
|
|
|
23,959 |
|
|
|
(1,725 |
) |
Stock-based compensation expenses |
|
|
7,854 |
|
|
|
9,732 |
|
|
|
(1,878 |
) |
Other expenses |
|
|
385 |
|
|
|
408 |
|
|
|
(23 |
) |
Sublicense and license fees |
|
|
1,660 |
|
|
|
798 |
|
|
|
862 |
|
Total research and development expenses |
|
$ |
68,574 |
|
|
$ |
72,484 |
|
|
$ |
(3,910 |
) |
The decrease of approximately $3.9 million was primarily attributable to a decrease in employee-related costs, including stock-based compensation expenses, offset by an increase in external research and development expenses.
General and Administrative Expenses
General and administrative expenses were $17.2 million for the three months ended March 31, 2026, compared to $19.3 million for the three months ended March 31, 2025. The decrease of approximately $2.1 million was primarily associated with a decrease in employee-related costs.
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Collaboration Expense, Net
Collaboration expense, net, was $45.9 million for the three months ended March 31, 2026, compared to $57.5 million for the three months ended March 31, 2025. The decrease was primarily attributable to an increase in our share of CASGEVY revenue.
Other Income, Net
Other income was $8.2 million for the three months ended March 31, 2026, compared to $13.5 million of income for the three months ended March 31, 2025. The decrease of approximately $5.3 million in other income was primarily due to the change in fair value of corporate equity securities during the three months ended March 31, 2026.
Liquidity and Capital Resources
We have predominantly incurred losses and cumulative negative cash flows from operations since our inception. As of March 31, 2026, we had $2,441.8 million in cash, cash equivalents and marketable securities, of which approximately $305.2 million was held outside of the United States, and an accumulated deficit of $2,070.5 million. We anticipate that we will continue to incur losses for at least the next several years. We expect to continue to incur research and development costs and general and administrative expenses consistent with costs associated with research and development at companies of our size and stage of development, and, as a result, we will need additional capital to fund our operations, which we may raise through public or private equity or debt financings, strategic collaborations, or other sources.
Debt
Notes, net
In March 2026, we issued $600.0 million aggregate principal amount of our Convertible Senior Notes due 2031, or the Notes, pursuant to an indenture dated March 16, 2026, or the Indenture, between us and U.S. Bank Trust Company, National Association, as trustee, in a private offering to qualified institutional buyers, or the 2026 Note Offering. The Notes issued in the 2026 Note Offering include the exercise in full of the initial purchasers’ option to purchase $50.0 million of additional Notes. The investors in the Notes agreed to an effective coupon of 1.125%. Because of anticipated 35% withholding on interest payments on the Notes under Swiss tax law, we agreed to increase the coupon by 0.6058% to 1.7308% to effectively eliminate the impact of such anticipated withholding on any holders who are not eligible to receive a refund.
The Notes are senior, unsecured obligations of ours and will mature on March 1, 2031, or the maturity date, unless earlier converted, redeemed or repurchased. The Notes will accrue interest payable semiannually in arrears on March 1 and September 1 of each year, beginning on September 1, 2026. Holders may convert all or any portion of their Notes at their option prior to the maturity date, other than during a “conversion freeze period” (as defined in the Indenture). Upon conversion, we will deliver our common shares, nominal value CHF 0.03 per share, or the common shares, at an initial conversion rate of 13.0617 common shares per $1,000 principal amount of Notes (equivalent to an initial conversion price of approximately $76.56 per common share), subject to adjustment in some events in accordance with the terms of the Indenture but will not be adjusted for any accrued and unpaid interest.
In addition, if certain corporate events occur or are anticipated to occur prior to the maturity date or if we deliver a notice of optional redemption, we will, in certain circumstances, increase the conversion rate for a holder who elects to convert our Notes in connection with such a corporate event or called (or deemed called) for redemption in connection with such notice of optional redemption. Initially, a maximum of 11,363,580 common shares may be delivered upon conversion of the Notes, based on the initial maximum conversion rate of 18.9393 common shares per $1,000 principal amount of Notes, which is subject to customary conversion rate adjustment provisions.
We may not redeem the Notes prior to March 6, 2029. On or after such date, we may redeem for cash all or any portion of the Notes (subject to the partial redemption limitation described in the Indenture), at our option, if the last reported sale price of our common shares has been at least 130% of the conversion price for the Notes then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending on, and including, the trading day immediately preceding the date on which we provide notice of optional redemption, at a redemption price equal to 100% of the principal amount of the Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the optional redemption date. No sinking fund is provided for the Notes.
If we undergo a fundamental change (as defined in the Indenture), then, subject to certain conditions and except as described in the Indenture, holders may require us to repurchase for cash all or any portion of their Notes at a fundamental change repurchase price equal to 100% of the principal amount of the Notes to be repurchased, plus accrued and unpaid interest to, but excluding, the fundamental change repurchase date.
The Indenture includes customary covenants and sets forth certain events of default after which the Notes may be declared immediately due and payable and sets forth certain types of bankruptcy or insolvency events of default involving us after which the Notes become automatically due and payable.
We received net proceeds from the 2026 Note Offering of approximately $585.4 million, after deducting the initial purchasers’ discounts and commissions and the estimated offering expenses payable by us. Refer to Note 8 to our condensed consolidated
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financial statements included in this Form 10-Q for other details related to the Notes.
At-the-Market Offerings (ATM)
We have entered into an Open Market Sale AgreementSM, or the Sales Agreement, with Jefferies LLC under which we, at our sole discretion, are able to offer and sell, from time to time at prevailing market prices, our common shares. The following are in connection with the Sales Agreement.
2021 ATM
In January 2021, we filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $600.0 million, or, together with the subsequent prospectus supplements filed in July 2021 and August 2024 relating to our common shares remaining under our original prospectus supplement, the 2021 ATM.
For the three months ended March 31, 2025, we issued and sold an aggregate of 0.2 million common shares under the 2021 ATM at an average price of $54.33 per share for aggregate proceeds of $8.7 million, which were net of equity issuance costs of $0.1 million, excluding stamp taxes of $0.1 million.
As of December 31, 2025, we had issued and sold an aggregate of 8.0 million common shares under the 2021 ATM at an average price of $74.77 per share for aggregate proceeds of $592.2 million, which were net of equity issuance costs of $7.8 million, excluding stamp taxes of $5.9 million. As of December 31, 2025, no common shares remained available under the 2021 ATM.
2025 ATM
In October 2025, we filed a new prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $600.0 million, or the 2025 ATM.
No common shares were issued and sold under the 2025 ATM for the three months ended March 31, 2026. As of March 31, 2026, we issued and sold an aggregate of 0.7 million common shares under the 2025 ATM at an average price of $60.81 per share for aggregate proceeds of $42.3 million, which were net of equity issuance costs of $0.5 million, excluding stamp taxes of $0.4 million. Common shares having aggregate gross proceeds up to $557.2 million remain available under the 2025 ATM.
Share Issuance Agreement with Sirius Therapeutics
As described in Note 6 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q, we and Sirius entered into a share issuance agreement in 2025 and we registered and issued 1,842,105 common shares to Sirius at an issue price of $38.00 per share as partial consideration for entering into the Sirius Agreement.
Funding Requirements
Our primary uses of capital are, and we expect will continue to be, research and development activities, manufacturing activities, compensation and employee-related expenses, laboratory and related supplies, legal and other regulatory expenses, patent prosecution, filing, defense and intellectual property maintenance costs, business development activities, and general overhead costs, including costs associated with operating as a public company. We expect to continue to incur operating expenses consistent with costs associated with research and development at companies of our size and stage of development, which may increase in the future to support continued research and development activities and potential commercialization of our product candidates.
Although we and our partner, Vertex, have received marketing approvals for CASGEVY in certain jurisdictions, we cannot guarantee we and Vertex will receive additional marketing approvals for CASGEVY or we will receive marketing approvals for our other product candidates in the future. Most of our programs are still in early stages of research and development and the outcome of our efforts is uncertain, and we cannot estimate the actual amounts necessary to successfully complete the development, manufacture and commercialization of any current or future product candidates, if approved, or whether, or when, we may achieve profitability. Until such time as we can generate substantial product revenues, if ever, we expect to finance our cash needs through a combination of equity financings, debt financings and payments received in connection with our collaboration and license agreements. We intend to consider opportunities to raise additional funds through the sale of equity or debt securities when market conditions are favorable to us to do so. However, the trading prices for our common shares and other biopharmaceutical companies have been highly volatile. As a result, we may face difficulties raising capital through sales of our common shares or such sales may be on unfavorable terms. In addition, a recession, depression or other sustained adverse market event, including resulting from the uncertain geopolitical environment, global trade policy and global economic conditions, could materially and adversely affect our business and the value of our common shares. To the extent that we raise additional capital through the future sale of equity or debt securities, the ownership interests of our shareholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing shareholders. If we raise additional funds through license or collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
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Outlook
Based on our research and development plans and our timing expectations related to the progress of our programs, we expect our existing cash, cash equivalents and marketable securities will enable us to fund our operating expenses and capital expenditures for at least the next 24 months without giving effect to any additional proceeds we may receive under our license agreements and collaborations, including with Vertex, and any other capital raising transactions we may complete. We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we expect. Given our need for additional financing to support the long-term clinical development and future commercialization of our programs, as applicable, we intend to consider additional financing opportunities when market terms are favorable to us.
Our ability to generate revenue and achieve profitability depends significantly on our success in many areas, including: developing our delivery technologies and our gene editing technology platform; selecting appropriate product candidates to develop; completing research and preclinical and clinical development of selected product candidates; obtaining regulatory approvals and marketing authorizations for product candidates for which we complete clinical trials; developing a sustainable and scalable manufacturing process for product candidates; launching and commercializing product candidates for which we obtain regulatory approvals and marketing authorizations, either directly or with a collaborator or distributor; obtaining market acceptance of our product candidates, either directly or with a collaborator or distributor, if approved, including for CASGEVY; addressing any competing technological and market developments; negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter; maintaining good relationships with our collaborators and licensors; maintaining, defending, protecting and expanding our estate of intellectual property rights, including patents, trade secrets and know-how; and attracting, hiring and retaining qualified personnel.
Cash Flows
The following table provides information regarding our cash flows for each of the periods below (in thousands):
|
|
Three Months Ended March 31, |
|
|
Period to Period |
|
||||||
|
|
2026 |
|
|
2025 |
|
|
Change |
|
|||
Net cash used in operating activities |
|
$ |
(108,897 |
) |
|
$ |
(53,947 |
) |
|
$ |
(54,950 |
) |
Net cash used in investing activities |
|
|
(410,743 |
) |
|
|
(19,755 |
) |
|
|
(390,988 |
) |
Net cash provided by financing activities |
|
|
591,908 |
|
|
|
10,588 |
|
|
|
581,320 |
|
Effect of exchange rate changes on cash |
|
|
(32 |
) |
|
|
41 |
|
|
|
(73 |
) |
Net increase (decrease) in cash |
|
$ |
72,236 |
|
|
$ |
(63,073 |
) |
|
$ |
135,309 |
|
Operating Activities
Net cash used in operating activities was $108.8 million for the three months ended March 31, 2026, compared to $53.9 million for the three months ended March 31, 2025. The $54.9 million increase in net cash used in operating activities was primarily driven by a $70.3 million overall decrease in net changes of operating assets and liabilities, primarily driven by the timing of receipt of a $25.0 million milestone payment from Vertex which was paid in the first quarter of 2025, offset by a decrease in net loss of approximately $13.1 million.
Investing Activities
Net cash used in investing activities for the three months ended March 31, 2026 was $410.7 million, compared to net cash used in investing activities of $19.8 million for the three months ended March 31, 2025. The increase in net cash used in investing activities was primarily driven by a net increase in purchases of our marketable securities.
Financing Activities
Net cash provided by financing activities for the three months ended March 31, 2026 was $591.9 million, compared with $10.6 million for the three months ended March 31, 2025. Net cash provided by financing activities for the three months ended March 31, 2026 consisted primarily of $585.4 million in net proceeds from the issuance of the Notes in March 2026.
Critical Accounting Policies and Estimates
There have been no material changes in our critical accounting policies and estimates in the preparation of our condensed consolidated financial statements during the three months ended March 31, 2026 compared to those discussed in our Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on February 12, 2026, with the exception of the new policy with respect to convertible debt described below.
Convertible debt
We account for our convertible debt instruments as a single unit of accounting, a liability, as we concluded that the conversion features do not require bifurcation as a derivative under ASC Topic 815-15, Derivatives and Hedging, as we did not issue our convertible debt instruments at a substantial premium. We record debt issuance costs as contra-liabilities in our condensed
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consolidated balance sheets at issuance and amortize debt issuance costs over the contractual term of the convertible debt instrument using the effective interest rate. The balance of our Notes presented in our condensed consolidated balance sheets represents the principal balance of the convertible debt instrument less unamortized debt issuance costs. Please refer to Note 8 of the notes to the condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for further information.
Recent Accounting Pronouncements
Refer to Note 1 of the notes to our unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for a discussion of recent accounting pronouncements.
Item 3. Quantitative and Qualitative Disclosures about Market Risk
Interest Rate Sensitivity
We are exposed to market risk related to changes in interest rates. As of March 31, 2026, we had cash, cash equivalents and marketable securities of $2,441.8 million, primarily invested in U.S. treasury securities and government agency securities, corporate bonds, commercial paper and money market accounts invested in U.S. government agency securities. Due to the conservative nature of these instruments, we do not believe that we have a material exposure to interest rate risk. If interest rates were to increase or decrease by 1%, the fair value of our investment portfolio would increase or decrease by an immaterial amount.
Foreign Currency Exchange Rate Risk
As a result of our foreign operations, we face exposure to movements in foreign currency exchange rates, primarily the Swiss Franc and British Pound, against the U.S. dollar. The current exposures arise primarily from cash, accounts payable and intercompany receivables and payables. Changes in foreign exchange rates affect our condensed consolidated statements of operations and distort comparisons between periods. To date, foreign currency transaction gains and losses have not been material to our financial statements, and we have not engaged in any foreign currency hedging transactions.
Inflation
Inflation generally affects us by increasing our cost of labor, clinical trial and manufacturing costs. We do not believe that inflation had a material effect on our business, financial condition or results of operations during the three months ended March 31, 2026 and 2025.
Item 4. Controls and Procedures.
Management’s Evaluation of our Disclosure Controls and Procedures
We maintain disclosure controls and procedures that are designed to ensure that information required to be disclosed in the reports that we file or submit under the Securities Exchange Act of 1934 is (1) recorded, processed, summarized, and reported within the time periods specified in the SEC’s rules and forms and (2) accumulated and communicated to our management, including our principal executive officer and principal financial officer, to allow timely decisions regarding required disclosure.
As of March 31, 2026, our management, with the participation of our principal executive officer and principal financial officer, evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934). Our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives, and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Our principal executive officer and principal financial officer have concluded based upon the evaluation described above that, as of March 31, 2026, our disclosure controls and procedures were effective at the reasonable assurance level.
Changes in Internal Control over Financial Reporting
During the quarter ended March 31, 2026, there were no changes in our internal control over financial reporting, as such term is defined in Rules 13a-15(f) and 15(d)-15(f) promulgated under the Securities Exchange Act of 1934, that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.
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PART II—OTHER INFORMATION
Item 1. Legal Proceedings.
In the ordinary course of business, we are or have been from time to time involved in lawsuits, investigations, proceedings and threats of litigation related to, among other things, our intellectual property estate (including certain in-licensed intellectual property), commercial arrangements and other matters. Such proceedings may include quasi-litigation, inter partes administrative proceedings in the U.S. Patent and Trademark Office and the European Patent Office involving our intellectual property estate including certain in-licensed intellectual property. For example, in the fourth quarter of 2025, ToolGen, Inc., or ToolGen, initiated a lawsuit against us and other third parties alleging patent infringement by CASGEVY of a ToolGen patent relating to CRISPR/Cas9 gene editing technology. We were subsequently dismissed from the lawsuit as a defendant in April 2026 without prejudice. The outcome of any of the foregoing is inherently uncertain. In addition, litigation and related matters are costly and may divert the attention of our management and other resources that would otherwise be engaged in other activities. If we were unable to prevail in any such proceedings, our business, results of operations, liquidity and financial condition could be adversely affected. There are currently no claims or actions pending against us that, in the opinion of our management, are likely to have a material adverse effect on our business.
There have been no material developments with respect to the legal proceedings previously disclosed in “Item 3. Legal Proceedings” of our Annual Report on Form 10-K for the fiscal year ended December 31, 2025 filed with the SEC on February 12, 2026.
Item 1A. Risk Factors.
We are updating and supplementing our risk factors previously disclosed in Item 1A of our Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on February 12, 2026 to add the following risk factors:
Our Indebtedness and Liabilities Could Limit the Cash Flow Available for Our Operations, Expose Us to Risks That Could Adversely Affect Our Business, Financial Condition and Results of Operations and Impair Our Ability to Satisfy Our Obligations Under the Notes.
In March 2026, we completed the 2026 Note Offering, issuing an aggregate principal amount of $600.0 million of Notes. This level of debt could have significant consequences on our future operations, including:
Any of the above-listed factors could have an adverse effect on our business, financial condition and results of operations and our ability to meet our payment obligations under the Notes and our other debt.
Our ability to meet our payment and other obligations under our debt instruments depends on our ability to generate significant cash flow in the future. This, to some extent, is subject to general economic, financial, competitive, legislative and regulatory factors as well as other factors that are beyond our control. We cannot assure you that our business will generate cash flow from operations, or that future borrowings will be available to us under our existing indebtedness or otherwise, in an amount sufficient to enable us to meet our payment obligations under the Notes and our other debt and to fund other liquidity needs. If we cannot generate sufficient cash flow to service our debt obligations, we may need to refinance or restructure our debt, including the Notes, sell assets, reduce or delay capital investments, seek to raise additional capital or any combination of the foregoing. If we raise additional debt, it would increase our interest expense, leverage and operating and financial costs. We cannot assure you that any of these actions could be effected on satisfactory terms, if at all, or that they would yield sufficient funds to make required payments on the Notes and any other indebtedness or to fund our other liquidity needs. In addition, the terms of existing or future debt agreements may restrict us from adopting any of these alternatives. We cannot assure you that our business will generate sufficient cash flows from operations or that future borrowings will be available in an amount sufficient to enable us to pay our indebtedness, including the Notes, or to fund our other liquidity needs. The failure to generate sufficient cash flow or to effect any of these alternatives could significantly adversely affect the value of the Notes and our ability to pay amounts due under the Notes.
Transactions Relating To Our Notes May Affect The Value Of Our Common Shares.
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The conversion of some or all of the Notes would dilute the ownership interests of existing shareholders to the extent we satisfy our conversion obligation by delivering common shares upon any conversion of such Notes. Our Notes may become in the future convertible at the option of their holders under certain circumstances. If holders of our Notes elect to convert their Notes, we may settle our conversion obligation by delivering to them a significant number of common shares, which would cause dilution to our existing shareholders.
There have been no other material changes to the risk factors previously disclosed in Part I, Item 1A (Risk Factors) of our Annual Report on Form 10-K for the fiscal year ended December 31, 2025 filed with the SEC on February 12, 2026. Please refer to the complete Part I, Item 1A of our Annual Report for additional risks and uncertainties we are facing that may have a material adverse effect on our business prospects, financial condition and results of operations. In addition to the risks described in our Annual Report on Form 10-K, you should carefully consider the other information set forth in this Form 10-Q and the information in our other filings with the SEC, as they could materially affect our business, financial condition or future results of operations.
Item 2. Unregistered Sales of Equity Securities and Use of Proceeds.
None.
Item 3. Defaults Upon Senior Securities.
Not applicable.
Item 4. Mine Safety Disclosures.
Not applicable.
Item 5. Other Information.
(a) Not applicable.
(b) Not applicable.
(c) From time to time, our officers (as defined in Rule 16a–1(f)) and directors may enter into Rule 10b5-1 or non-Rule 10b5-1 trading arrangements (as each such term is defined in Item 408 of Regulation S-K). During the three months ended March 31, 2026, none of our officers and directors
Item 6. Exhibits
The exhibits filed as part of this Quarterly Report on Form 10-Q are set forth in the Exhibit Index below.
Exhibit Number |
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Description of Document |
4.1 |
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Indenture, dated as of March 16, 2026, by and between the Company and U.S. Bank Trust Company, National Association, as Trustee (incorporated herein by reference to Exhibit 4.1 to the Company’s Current Report on Form 8-K filed on March 16, 2026) |
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4.2 |
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Form of Global Note, representing the Company’s Convertible Senior Notes due 2031 (included as Exhibit A to the Indenture filed as Exhibit 4.1 to the Company’s Current Report on Form 8-K filed on March 16, 2026) (incorporated herein by reference to Exhibit 4.2 to the Company’s Current Report on Form 8-K filed on March 16, 2026) |
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31.1* |
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Certification of Chief Executive Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 |
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31.2* |
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Certification of Chief Financial Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 |
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32.1*+ |
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Certification pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002 |
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101.INS* |
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Inline XBRL Instance Document – the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the inline XBRL document |
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101.SCH* |
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Inline XBRL Taxonomy Extension Schema With Embedded Linkbase Documents |
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104* |
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Cover Page Interactive Data File (formatted as inline XBRL with applicable taxonomy extension information contained in Exhibits 101) |
* Filed herewith.
+ The certification attached as Exhibit 32.1 that accompany this Quarterly Report on Form 10-Q are not deemed filed with the Securities and Exchange Commission and are not to be incorporated by reference into any filing of CRISPR Therapeutics AG under the
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Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date of this Form 10-Q, irrespective of any general incorporation language contained in such filing.
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
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CRISPR Therapeutics AG |
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Dated: May 4, 2026 |
By: |
/s/ Samarth Kulkarni |
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Samarth Kulkarni, Ph.D. |
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Chief Executive Officer |
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(Principal Executive Officer) |
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Dated: May 4, 2026 |
By: |
/s/ Raju Prasad |
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Raju Prasad, Ph.D. |
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Chief Financial Officer |
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(Principal Financial Officer) |
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